Company lists · Data refreshed September 2026

Top 25 Spinal muscular atrophy companies worldwide

RevenueBase identified 75 spinal muscular atrophy companies worldwide as of September 2026 and tracks 3,300 professional contacts across them, including 2,327 with verified work emails. The United States is the largest market and Cambridge the largest hub.

This list covers spinal muscular atrophy companies worldwide, 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. Country and city lists for spinal muscular atrophy companies follow the table.

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.

How to read this list HQ checked on every row RevenueBase Smart Search rating on every row Refreshed Sep 22, 2026 86% judged on-target in blind review
75Companies
3,300Contacts tracked
2,327Verified emails
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The 25 largest spinal muscular atrophy companies worldwide

Ranked by team size
Top 25 of 75 spinal muscular atrophy companies worldwide, ranked by team size, largest first. Data refreshed Sep 22, 2026. RevenueBase Smart Search rates how each company's information relates to spinal muscular atrophy companies: strong evidence (its own description names it), partial (names part of it), indirect (matched on the rest of its profile rather than the description shown). Click a contact count to view that company's people.
#CompanyRevenueBase Smart SearchHeadquartersEmployeesContacts
1Indirect evidenceCambridge, MA, United StatesHQ checked Sep 22, 2026501-1,0001,359 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, 2026
2Indirect evidenceCambridge, MA, United StatesHQ checked Sep 22, 202651-200325 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, 2026
3Indirect evidenceWaltham, MA, United StatesHQ checked Sep 22, 202651-200256 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, 2026
4Indirect evidenceBoston, MA, United StatesHQ checked Sep 22, 202651-200239 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, 2026
5Indirect evidenceBoston, MA, United StatesHQ checked Sep 22, 202651-200160 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, 2026
6Indirect evidenceParamus, NJ, United StatesHQ checked Sep 22, 202651-200155 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, 2026
7Indirect evidenceBoulder, CO, United StatesHQ checked Sep 22, 202651-200142 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, 2026
8Indirect evidencePratteln, SwitzerlandHQ checked Sep 22, 202651-200113 contacts →

Indirect evidence

“Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular…”

Source: RevenueBase Database · checked Sep 22, 2026
9Strong evidenceSchaumburg, IL, United StatesHQ checked Sep 22, 202611-5077 contacts →

Strong evidence

“Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA).”

Source: RevenueBase Database · checked Sep 22, 2026
10Indirect evidencePhiladelphia, PA, United StatesHQ checked Sep 22, 202651-20056 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, 2026
11Indirect evidenceUnited StatesHQ checked Sep 22, 20261,001-5,00035 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, 2026
12Indirect evidenceChapel Hill, NC, United StatesHQ checked Sep 22, 202611-5034 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, 2026
13Indirect evidenceSeoul, South KoreaHQ checked Sep 22, 202651-20033 contacts →

Indirect evidence

“Helixmith is a gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals for previously untreated diseases, and is listed on KOSDAQ.”

Source: RevenueBase Database · checked Sep 22, 2026
14Indirect evidenceNY, United StatesHQ checked Sep 22, 202651-20029 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, 2026
15Strong evidenceFreiburg, GermanyHQ checked Sep 22, 20261-1015 contacts →

Strong evidence

“SMA Europe is a non-profit umbrella organisation of spinal muscular atrophy (SMA) patient organisations from across Europe.”

Source: RevenueBase Database · checked Sep 22, 2026
16Strong evidenceStratford-upon-avon, United KingdomHQ checked Sep 22, 202611-5014 contacts →

Strong evidence

“SMA UK is a national charity supporting anyone affected by spinal muscular atrophy, a rare genetic, neuromuscular condition that causes muscle weakness and loss of movement.”

Source: RevenueBase Database · checked Sep 22, 2026
17Indirect evidenceSan Diego, CA, United StatesHQ checked Sep 22, 202611-5012 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, 2026
18Indirect evidenceDurham, NC, United StatesHQ checked Sep 22, 20261-1011 contacts →

Indirect evidence

“AMO Pharma is a biopharmaceutical company incorporated in February of 2015.”

Source: RevenueBase Database · checked Sep 22, 2026
19Strong evidenceWarsaw, PolandHQ checked Sep 22, 202611-5010 contacts →

Strong evidence

“SMA Foundation is a Warsaw, Poland based patient organisation that works with all those affected by spinal muscular atrophy (SMA).”

Source: RevenueBase Database · checked Sep 22, 2026
20Indirect evidenceMelbourne, AustraliaHQ checked Sep 22, 20261-1010 contacts →

Indirect evidence

“Percheron Therapeutics Limited (ASX: PER) is an international biotechnology company focused on the development of novel therapies for rare diseases.”

Source: RevenueBase Database · checked Sep 22, 2026
21Indirect evidenceNew York, NY, United StatesHQ checked Sep 22, 20261-1010 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, 2026
22Indirect evidenceWendelsheim, GermanyHQ checked Sep 22, 202611-508 contacts →

Indirect evidence

“MODAG focuses on research and development of therapeutics and diagnostics for neurodegenerative diseases.”

Source: RevenueBase Database · checked Sep 22, 2026
23Strong evidenceZbuzany, Czech RepublicHQ checked Sep 22, 20261-108 contacts →

Strong evidence

“SMÁci is the patient organisation for Spinal Muscular Atrophy patients in the Czech Republic.”

Source: RevenueBase Database · checked Sep 22, 2026
24Indirect evidenceNew York, NY, United StatesHQ checked Sep 22, 202611-507 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, 2026
25Partial evidenceBadalona, SpainHQ checked Sep 22, 20261-106 contacts →

Partial evidence

“Biointaxis is an academic spin-off focused on the development of gene therapies for neurodegenerative disorders.”

Source: RevenueBase Database · checked Sep 22, 2026

Showing 25 of 75 spinal muscular atrophy companies worldwide

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Spinal muscular atrophy companies worldwide at a glance

As of Sep 22, 2026
70.5%Verified-email coverage
44.0Average contacts per company
1-10Median size (employees)
5.3%Largest-city concentration · Cambridge
Sep 22, 2026Page data refreshed

Companies by employee count

1-10
49
11-50
12
51-200
11
501-1,000
1
1,001-5,000
2

Email recency is reported on each country list linked below.

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.
Coverage
Every country. Country and city lists for spinal muscular atrophy companies are linked below.
What qualifies
Companies worldwide 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.
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, 2,327 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.

Spinal muscular atrophy companies by location

1 lists · largest first

Every Spinal muscular atrophy companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.

Countries

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 worldwide?

RevenueBase identified 75 spinal muscular atrophy companies worldwide 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 country has the most spinal muscular atrophy companies?

The United States leads with 34 companies. Every spinal muscular atrophy companies list by country and city is linked on this page.

Which city has the most spinal muscular atrophy companies?

Cambridge leads with 4 headquartered companies, followed by Sao Paulo (3) and Boston (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 2,327 verified decision-maker contacts tracked across these 75 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.