Company lists · Data refreshed September 2026
Top 23 Spinal muscular atrophy companies based in the United States
RevenueBase identified 34 spinal muscular atrophy companies headquartered in the United States as of September 2026 and tracks 2,975 professional contacts across them, including 2,184 with verified work emails. Cambridge is the largest hub.
This list covers spinal muscular atrophy companies headquartered in the United States, matched on what each company actually does as described in its own words. Ranked by team size, largest first. Every row shows the company's own description, its RevenueBase Smart Search rating, and when it was last checked. Data refreshed September 22, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase Smart Search, 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 23 largest spinal muscular atrophy companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 1,359 contacts → | ||||
Indirect evidence “Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 2 | 325 contacts → | ||||
Indirect evidence “Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 3 | 256 contacts → | ||||
Indirect evidence “Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 4 | 239 contacts → | ||||
Indirect evidence “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…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 5 | 160 contacts → | ||||
Indirect evidence “Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 6 | 155 contacts → | ||||
Indirect evidence “NS Pharma, Inc.(NSP) is a biopharmaceutical leader in delivering life-changing care to people living with complex, rare diseases, backed by over 100 years of Nippon Shinyaku Co.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 7 | 142 contacts → | ||||
Indirect evidence “Edgewise Therapeutics, Inc.(Nasdaq: EWTX) is a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 8 | 77 contacts → | ||||
Strong evidence “Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA).” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 9 | 56 contacts → | ||||
Indirect evidence “GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 10 | 35 contacts → | ||||
Indirect evidence “Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 11 | 34 contacts → | ||||
Indirect evidence “Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 12 | 29 contacts → | ||||
Indirect evidence “At Prevail Therapeutics, we are developing novel gene therapies that target the root genetic causes of Parkinson’s disease and other neurodegenerative disorders.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 13 | 12 contacts → | ||||
Indirect evidence “Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 14 | 11 contacts → | ||||
Indirect evidence “AMO Pharma is a biopharmaceutical company incorporated in February of 2015.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 15 | 10 contacts → | ||||
Indirect evidence “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.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 16 | 7 contacts → | ||||
Indirect evidence “Aruvant’s mission is to bring hope to patients living with rare diseases by developing life changing and potentially curative gene therapies, with a near-term focus on sickle cell…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 17 | 5 contacts → | ||||
Indirect evidence “Mission: Transforming lives and building a kinder and more inclusive future through advocacy, research, and support.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 18 | 5 contacts → | ||||
Indirect evidence “Sarcomatrix, Inc.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 19 | 3 contacts → | ||||
Indirect evidence “The Cole Parker Randall Foundation was established in 2014 by Cole's parents Dave and Shawna Randall and their children Jase and Cade Randall, Cole's older brothers.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 20 | 2 contacts → | ||||
Indirect evidence “Orphina is a pre-startup biotechnology company focused on preserving and prolonging muscle health.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 21 | 2 contacts → | ||||
Indirect evidence “Genea Biocells is a neuromuscular disease-focused company based on a proprietary technology platform for the accelerated development of drugs to treat neuromuscular diseases -…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 22 | 2 contacts → | ||||
Indirect evidence “Dystrogen Therapeutics is a clinical stage chimeric cell therapy company focusing on rare diseases.” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
| 23 | 2 contacts → | ||||
Partial evidence “Shift Pharmaceuticals is developing Antisense Oligonucleotides as drug candidates for a variety of genetic disorders, including Charcot Marie Tooth Syndrome (CMT1A) and Spinal…” Source: RevenueBase Database · checked Sep 22, 2026Thanks. The Data Team will review it. | |||||
RevenueBase Smart Search
RevenueBase Smart Search assesses information from a company’s LinkedIn profile, information supplied directly by the company, its website, and other publicly available sources. It uses that information to rate how closely the company relates to this list’s category: spinal muscular atrophy companies. Every row carries its rating and the description the rating was read from, so each one can be checked rather than taken on trust.
- Strong evidence
- The company's own public description of its business names spinal muscular atrophy companies.
- Partial evidence
- Its own public description names part of spinal muscular atrophy companies, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name spinal muscular atrophy companies. The match rests on the rest of the company's public profile rather than the one sentence quoted here, so this is the rating to check first.
RevenueBase Smart Search is how these lists are built: RevenueBase's data engine and AI assemble the companies that match a category defined in a user's own words, so a list can be about what companies actually do rather than about whichever industry code they were filed under.
Showing 23 of 34 spinal muscular atrophy companies based in the United States
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Spinal muscular atrophy 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 34 companies.
How this list was built
Company categories are rarely black and white. RevenueBase reads each company's own description of its business to decide whether it belongs among spinal muscular atrophy companies, so some rows will be adjacent to the category rather than pure plays. The description and RevenueBase Smart Search rating on every row are there so you can check each match yourself.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase Smart Search, the natural-language company search available to every RevenueBase user.
- What qualifies
- Companies headquartered in the United States whose own description of what they do matches “spinal muscular atrophy companies”. Matching reads each company's description, not its industry code or its name.
- RevenueBase Smart Search
- RevenueBase Smart Search is how this list is assembled and how each row is rated. RevenueBase's data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to a category defined in a user's own words. Each row is rated strong evidence (its own description names spinal muscular atrophy companies), partial evidence (it names part of the category) or indirect evidence (the description shown names none of it and the match rests on the rest of the company's profile). The description supporting each rating is quoted on the row.
- What we exclude
- Companies whose own description does not clearly place them among spinal muscular atrophy companies, and records with no verified work email. Companies headquartered outside United States are excluded even when they keep an office there.
- Match quality
- In blind human judging of this matching method, 86% of matched companies were on target. Expect roughly 1 in 7 rows to be adjacent to the category rather than a pure play. Every row shows the company's own description and its RevenueBase Smart Search rating so you can judge each match yourself. Read the full methodology: how the lists are built, how the judging worked, and what the figure means.
- 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 listed company carries at least one verified work email, 2,184 across the full list. 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.
- Corrections
- Wrong category, wrong headquarters, or a company that has closed? Use “Report an issue” on any row. Reports go to the RevenueBase Data Team and feed back into the company graph.
Frequently asked
What is RevenueBase Smart Search?
RevenueBase Smart Search is how RevenueBase decides which companies belong on this list and how strongly each one fits. Its data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to the category the list is about. Each row is rated strong evidence when the company's own description names spinal muscular atrophy companies, partial evidence when it names part of the category, and indirect evidence when the description shown names none of it and the match rests on the rest of the company's profile.
How many spinal muscular atrophy companies are there based in the United States?
RevenueBase identified 34 spinal muscular atrophy companies headquartered in the United States as of September 2026, matched on each company's own description of what it does rather than its industry code.
What is the largest company on this list?
Sarepta Therapeutics, headquartered in Cambridge, tops this list with the largest team on record.
Which city has the most spinal muscular atrophy companies?
Cambridge leads with 4 headquartered companies, followed by Boston (2) and New York (2).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work the top results, verified emails and direct dials included. Upgrading unlocks all 2,184 verified decision-maker contacts tracked across these 34 companies.
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.
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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 Smart Search, 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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