Company lists · Data refreshed September 2026
Top 19 Spinal muscular atrophy therapy companies based in the United States
RevenueBase identified 35 company matches for spinal muscular atrophy therapy companies associated with the United States as of September 2026 and tracks 3,079 professional contacts across them, including 2,337 with verified work emails. Cambridge is the largest hub.
This list covers company matches for spinal muscular atrophy therapy companies associated with the United States, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 22, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.
The 19 largest spinal muscular atrophy therapy companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 1,359 contacts → | ||||
Exact match “Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 2 | 510 contacts → | ||||
Exact match “AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 3 | 325 contacts → | ||||
Exact match “Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 4 | 256 contacts → | ||||
Exact match “Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 5 | 239 contacts → | ||||
Exact match “Headquartered in Boston’s Seaport community, Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 6 | 142 contacts → | ||||
Exact match “Edgewise Therapeutics, Inc.(Nasdaq: EWTX) is a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 7 | 77 contacts → | ||||
Exact match “Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA).” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 8 | 35 contacts → | ||||
Exact match “Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 9 | 34 contacts → | ||||
Exact match “Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 10 | 12 contacts → | ||||
Exact match “Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 11 | 10 contacts → | ||||
Exact match “The mission of the SMA Foundation is to accelerate the development of a treatment for SMA, the number one genetic killer of infants and toddlers.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 12 | 6 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 13 | 5 contacts → | ||||
Exact match “Sarcomatrix, Inc.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 14 | 5 contacts → | ||||
Exact match “Mission: Transforming lives and building a kinder and more inclusive future through advocacy, research, and support.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 15 | 2 contacts → | ||||
Exact match “Orphina is a pre-startup biotechnology company focused on preserving and prolonging muscle health.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 16 | 2 contacts → | ||||
Exact match “Genea Biocells is a neuromuscular disease-focused company based on a proprietary technology platform for the accelerated development of drugs to treat neuromuscular diseases -…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 17 | 2 contacts → | ||||
Exact match “Shift Pharmaceuticals is developing Antisense Oligonucleotides as drug candidates for a variety of genetic disorders, including Charcot Marie Tooth Syndrome (CMT1A) and Spinal…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 18 | 2 contacts → | ||||
Exact match “Dystrogen Therapeutics is a clinical stage chimeric cell therapy company focusing on rare diseases.” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
| 19 | 1 contacts → | ||||
Exact match “Papillon Therapeutics is a biotechnology company advancing genetic medicines for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease…” Recorded in the RevenueBase company graph · Sep 22, 2026 · View company records in RevenueBase | |||||
RevenueBase SmartSearch
Describe a niche business in your own words. RevenueBase SmartSearch compares the meaning of your description with company business descriptions compiled in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit.
This list uses Exact for “spinal muscular atrophy therapy companies”. Each Exact match badge shows membership in that saved search; it is not an individual similarity score or a service-quality rating. Recorded descriptions are shown where available.
Showing 19 of 35 spinal muscular atrophy therapy companies based in the United States
See the full list, filter by size, metro and specialty, and access 2,337 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Spinal muscular atrophy therapy companies based in the United States at a glance
As of Sep 22, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 35 companies.
How this list was built
The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
- What qualifies
- Company records associated with the United States whose business descriptions match “spinal muscular atrophy therapy companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. Where headquarters information is recorded, preview rows are also filtered to this page’s location. Dataset totals and geographic summaries describe the full matched records before these preview filters.
- Match quality
- RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
- Ranking
- Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
- Verification
- Every displayed company carries at least one verified work email. RevenueBase tracks 2,337 professional contacts with verified work emails across the full matched dataset. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
- Refresh
- This page's figures were pulled from the company graph on September 22, 2026. Lists refresh periodically as the graph is re-checked.
- Record context
- Company records and descriptions can change. Check the company's website for its current services and locations.
Frequently asked
What is RevenueBase SmartSearch?
RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “spinal muscular atrophy therapy companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many spinal muscular atrophy therapy companies are there based in the United States?
RevenueBase identified 35 company matches for spinal muscular atrophy therapy companies associated with the United States as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.
What is the largest company on this list?
Sarepta Therapeutics tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which city has the most spinal muscular atrophy therapy companies?
Cambridge leads with 4 company records, followed by New York (2) and San Diego (2).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work limited free results with 500 credits to start. Upgrading provides access to the 2,337 professional contacts with verified work emails tracked across these 35 companies; direct dials are available where recorded.
How current is this list?
The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 22, 2026.
Ready to reach these companies? Verified emails and direct dials for all 35 spinal muscular atrophy therapy companies based in the United States. Start with a free account, no credit card.
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About this data
RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase SmartSearch, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.
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Company intelligence · public preview
Top 19 spinal muscular atrophy therapy companies based in the United States
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 35 matching company records for spinal muscular atrophy therapy companies based in the United States, with 3,079 tracked contacts and 2,337 verified work emails.
This public preview shows 19 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 22, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 19 spinal muscular atrophy therapy companies based in the United States
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 |  Sarepta Therapeuticssarepta.comHQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 1,359 contacts → |
“Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  Askbio INCaskbio.comHQ: Research Triangle Park, NC, United StatesReported employees: 501-1,000 | Research Triangle Park, NC, United States | 501-1,000 | Exact match | 510 contacts → |
“AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  Scholar Rockscholarrock.comHQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 325 contacts → |
“Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Dyne Therapeuticsdyne-tx.com HQ: Waltham, MA, United StatesReported employees: 51-200 | Waltham, MA, United States | 51-200 | Exact match | 256 contacts → |
“Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Entrada Therapeuticsentradatx.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 239 contacts → |
“Headquartered in Boston’s Seaport community, Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Edgewise Therapeuticsedgewisetx.com HQ: Boulder, CO, United StatesReported employees: 51-200 | Boulder, CO, United States | 51-200 | Exact match | 142 contacts → |
“Edgewise Therapeutics, Inc.(Nasdaq: EWTX) is a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Cure SMAcuresma.org HQ: Schaumburg, IL, United StatesReported employees: 11-50 | Schaumburg, IL, United States | 11-50 | Exact match | 77 contacts → |
“Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA).” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  Novartis Gene Therapiesnovartis.comHQ: United StatesReported employees: 1,001-5,000 | United States | 1,001-5,000 | Exact match | 35 contacts → |
“Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Bamboo Therapeutics, Inc. A Wholly Owned Subsidiary Of Pfizer INCbambootherapeutics.com HQ: Chapel Hill, NC, United StatesReported employees: 11-50 | Chapel Hill, NC, United States | 11-50 | Exact match | 34 contacts → |
“Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | Epirium BIOepirium.com HQ: San Diego, CA, United StatesReported employees: 11-50 | San Diego, CA, United States | 11-50 | Exact match | 12 contacts → |
“Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | SMA Foundationsmafoundation.org HQ: New York, NY, United StatesReported employees: 1-10 | New York, NY, United States | 1-10 | Exact match | 10 contacts → |
“The mission of the SMA Foundation is to accelerate the development of a treatment for SMA, the number one genetic killer of infants and toddlers.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Armatus Bio, INCarmatusbio.com HQ: Columbus, OH, United StatesReported employees: 1-10 | Columbus, OH, United States | 1-10 | Exact match | 6 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 13 | Sarcomatrixsarcomatrix.com HQ: Reno, NV, United StatesReported employees: 1-10 | Reno, NV, United States | 1-10 | Exact match | 5 contacts → |
“Sarcomatrix, Inc.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Gwendolyn Strong Foundationnevergiveup.org HQ: Santa Barbara, CA, United StatesReported employees: 1-10 | Santa Barbara, CA, United States | 1-10 | Exact match | 5 contacts → |
“Mission: Transforming lives and building a kinder and more inclusive future through advocacy, research, and support.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Orphina Biotechnologyorphina.com HQ: Calabasas, CA, United StatesReported employees: 1-10 | Calabasas, CA, United States | 1-10 | Exact match | 2 contacts → |
“Orphina is a pre-startup biotechnology company focused on preserving and prolonging muscle health.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Genea Biocells Us, INCgeneabiocells.com HQ: La Jolla, CA, United StatesReported employees: 11-50 | La Jolla, CA, United States | 11-50 | Exact match | 2 contacts → |
“Genea Biocells is a neuromuscular disease-focused company based on a proprietary technology platform for the accelerated development of drugs to treat neuromuscular diseases -…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Shift Pharmaceuticalsshiftpharmaceuticals.com HQ: Overland Park, KS, United StatesReported employees: 1-10 | Overland Park, KS, United States | 1-10 | Exact match | 2 contacts → |
“Shift Pharmaceuticals is developing Antisense Oligonucleotides as drug candidates for a variety of genetic disorders, including Charcot Marie Tooth Syndrome (CMT1A) and Spinal…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Dystrogen Therapeuticsdystrogen.com HQ: Chicago, United StatesReported employees: 1-10 | Chicago, United States | 1-10 | Exact match | 2 contacts → |
“Dystrogen Therapeutics is a clinical stage chimeric cell therapy company focusing on rare diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Papillon Therapeuticspapillon-tx.com HQ: San Diego, CA, United StatesReported employees: 1-10 | San Diego, CA, United States | 1-10 | Exact match | 1 contacts → |
“Papillon Therapeutics is a biotechnology company advancing genetic medicines for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 19 of 35 company matches
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Spinal muscular atrophy therapy companies: dataset insights
September 22, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
35 records in the displayed employee bands.
0 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 35 companies.
How this company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 22, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Questions about this company list
What does Exact match mean for spinal muscular atrophy therapy companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many spinal muscular atrophy therapy companies did RevenueBase find based in the United States?
The saved snapshot records 35 matching company records, 3,079 tracked contacts and 2,337 verified emails. The public preview displays 19 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 22, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
Cite this list
RevenueBase Data Team. Top 19 spinal muscular atrophy therapy companies based in the United States. Snapshot: September 22, 2026. https://revenuebase.ai/companies/spinal-muscular-atrophy-therapy-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
- All company lists
- Lists by location
- Lists A-Z
- New lists
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
- Oil and gas companies based in the United States
- Real estate companies based in the United States
Company intelligence · public preview
Top 19 spinal muscular atrophy therapy companies based in the United States
RevenueBase records 35 matching company records for spinal muscular atrophy therapy companies based in the United States, with 3,079 tracked contacts and 2,337 verified work emails.
This public preview shows 19 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 22, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 19 spinal muscular atrophy therapy companies based in the United States
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 | Sarepta Therapeuticssarepta.com HQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 1,359 contacts → |
“Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | Askbio INCaskbio.com HQ: Research Triangle Park, NC, United StatesReported employees: 501-1,000 | Research Triangle Park, NC, United States | 501-1,000 | Exact match | 510 contacts → |
“AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | Scholar Rockscholarrock.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 325 contacts → |
“Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Dyne Therapeuticsdyne-tx.com HQ: Waltham, MA, United StatesReported employees: 51-200 | Waltham, MA, United States | 51-200 | Exact match | 256 contacts → |
“Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Entrada Therapeuticsentradatx.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 239 contacts → |
“Headquartered in Boston’s Seaport community, Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Edgewise Therapeuticsedgewisetx.com HQ: Boulder, CO, United StatesReported employees: 51-200 | Boulder, CO, United States | 51-200 | Exact match | 142 contacts → |
“Edgewise Therapeutics, Inc.(Nasdaq: EWTX) is a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Cure SMAcuresma.org HQ: Schaumburg, IL, United StatesReported employees: 11-50 | Schaumburg, IL, United States | 11-50 | Exact match | 77 contacts → |
“Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA).” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Novartis Gene Therapiesnovartis.com HQ: United StatesReported employees: 1,001-5,000 | United States | 1,001-5,000 | Exact match | 35 contacts → |
“Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Bamboo Therapeutics, Inc. A Wholly Owned Subsidiary Of Pfizer INCbambootherapeutics.com HQ: Chapel Hill, NC, United StatesReported employees: 11-50 | Chapel Hill, NC, United States | 11-50 | Exact match | 34 contacts → |
“Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | Epirium BIOepirium.com HQ: San Diego, CA, United StatesReported employees: 11-50 | San Diego, CA, United States | 11-50 | Exact match | 12 contacts → |
“Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | SMA Foundationsmafoundation.org HQ: New York, NY, United StatesReported employees: 1-10 | New York, NY, United States | 1-10 | Exact match | 10 contacts → |
“The mission of the SMA Foundation is to accelerate the development of a treatment for SMA, the number one genetic killer of infants and toddlers.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Armatus Bio, INCarmatusbio.com HQ: Columbus, OH, United StatesReported employees: 1-10 | Columbus, OH, United States | 1-10 | Exact match | 6 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 13 | Sarcomatrixsarcomatrix.com HQ: Reno, NV, United StatesReported employees: 1-10 | Reno, NV, United States | 1-10 | Exact match | 5 contacts → |
“Sarcomatrix, Inc.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Gwendolyn Strong Foundationnevergiveup.org HQ: Santa Barbara, CA, United StatesReported employees: 1-10 | Santa Barbara, CA, United States | 1-10 | Exact match | 5 contacts → |
“Mission: Transforming lives and building a kinder and more inclusive future through advocacy, research, and support.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Orphina Biotechnologyorphina.com HQ: Calabasas, CA, United StatesReported employees: 1-10 | Calabasas, CA, United States | 1-10 | Exact match | 2 contacts → |
“Orphina is a pre-startup biotechnology company focused on preserving and prolonging muscle health.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Genea Biocells Us, INCgeneabiocells.com HQ: La Jolla, CA, United StatesReported employees: 11-50 | La Jolla, CA, United States | 11-50 | Exact match | 2 contacts → |
“Genea Biocells is a neuromuscular disease-focused company based on a proprietary technology platform for the accelerated development of drugs to treat neuromuscular diseases -…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Shift Pharmaceuticalsshiftpharmaceuticals.com HQ: Overland Park, KS, United StatesReported employees: 1-10 | Overland Park, KS, United States | 1-10 | Exact match | 2 contacts → |
“Shift Pharmaceuticals is developing Antisense Oligonucleotides as drug candidates for a variety of genetic disorders, including Charcot Marie Tooth Syndrome (CMT1A) and Spinal…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Dystrogen Therapeuticsdystrogen.com HQ: Chicago, United StatesReported employees: 1-10 | Chicago, United States | 1-10 | Exact match | 2 contacts → |
“Dystrogen Therapeutics is a clinical stage chimeric cell therapy company focusing on rare diseases.” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Papillon Therapeuticspapillon-tx.com HQ: San Diego, CA, United StatesReported employees: 1-10 | San Diego, CA, United States | 1-10 | Exact match | 1 contacts → |
“Papillon Therapeutics is a biotechnology company advancing genetic medicines for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease…” Recorded description excerpt · September 22, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 19 of 35 company matches
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Spinal muscular atrophy therapy companies: dataset insights
September 22, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
35 records in the displayed employee bands.
0 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 35 companies.
How this company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 22, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Questions about this company list
What does Exact match mean for spinal muscular atrophy therapy companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many spinal muscular atrophy therapy companies did RevenueBase find based in the United States?
The saved snapshot records 35 matching company records, 3,079 tracked contacts and 2,337 verified emails. The public preview displays 19 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 22, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
Cite this list
RevenueBase Data Team. Top 19 spinal muscular atrophy therapy companies based in the United States. Snapshot: September 22, 2026. https://revenuebase.ai/companies/spinal-muscular-atrophy-therapy-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
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