Spinal Muscular Atrophy Foundation

What Spinal Muscular Atrophy Foundation does

Cure SMA, legally the Spinal Muscular Atrophy Foundation, is a 501(c)(3) nonprofit organization founded in 1984 and headquartered in Schaumburg, Illinois, that operates as the leading U.S.-anchored patient advocacy and research-funding body for Spinal Muscular Atrophy. The foundation coordinates a national network of local chapters, an expanding Care Center Network (10+ sites), the SMA Industry Collaboration that brings together major SMA pharmaceutical developers, and the SMA Drug Pipeline — a tracker now covering 18+ development programs. It funds basic, translational, and clinical research through grants ranging from $50,000 to $300,000 awarded to academic investigators, and has contributed to four FDA-approved SMA treatments: Spinraza (2016), Zolgensma (2019), Evrysdi (2020), and Itvisma. The organization also delivers patient and family services free of charge, including Care Series Booklets, the Compass research publication, the Directions community newsletter, conference scholarships (900+ annually for the Annual SMA Conference), and programs such as Walk-n-Roll, Summit of Strength, and virtual webinars and socials.

The foundation's revenue model is composed entirely of philanthropic streams: individual donations (including monthly giving and planned giving vehicles such as charitable remainder trusts and IRA rollovers), corporate and foundation grants from pharmaceutical partners (Biogen, Novartis Gene Therapies, Roche/Genentech, Cytokinetics) and charitable foundations (notably the Luke 18:1 Foundation with a $500,000 gift), event fundraising (Walk-n-Roll, Hope on the Hill Congressional Dinner, galas, endurance events, Stream Away SMA), and individual major gifts. Services and resources are provided free of charge to the SMA community, so the foundation is measured by programmatic output and impact milestones rather than transactional revenue. Operational scale is mid-sized (11–50 employees), and the organization operates with a board of directors and advisory bodies including a Medical Advisory Council and Adult Advisory Council, led by Kenneth Hobby as President following the retirement of Dr. Mary Schroth from the Chief Medical Officer role.

Name

Spinal Muscular Atrophy Foundation

Legal name

Cure SMA

Website

https://smafoundation.org

Company type

Private

Founded year

2003

Operating status

Operating

Headcount range

11–50 employees

Short description

Cure SMA (Spinal Muscular Atrophy Foundation) is a 501(c)(3) nonprofit founded in 1984 that funds SMA research, supports affected families, and coordinates industry collaboration, having contributed to four FDA-approved SMA treatments and 100% U.S. newborn screening coverage.

Ownership category

akta.pro rank

Product category

Disease-focused nonprofit patient advocacy and research funding

NAICS

Voluntary Health Organizations (813212), Social Assistance (624), Individual and Family Services (6241), Scientific Research and Development Services (5417)

SIC

Services-Social Services (8300), Services-Health Services (8000), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Site Network Operators & SMOs (HLAGACAA)

Keywords

• Rare disease research
• Patient advocacy services
• Medical research grants
• Patient community support
• Nonprofit healthcare foundation

Headquarters

HQ city

Illinois City

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Operations, Technology or R&D, Marketing or Sales, Others

Revenue model

1. Individual Donations: Charitable contributions from individuals, families, and supporters of the SMA community. The organization runs annual campaigns, monthly giving programs, and end-of-year fundraising appeals.
2. Corporate and Foundation Grants: Funding from pharmaceutical companies (Biogen, Roche, Novartis Gene Therapies), healthcare companies, and charitable foundations including Luke 18:1 Foundation, Miller McNeil Woodruff Foundation, and others.
3. Event Fundraising: Revenue from fundraising events including Walk-n-Roll, Annual SMA Conference, Hope on the Hill Congressional Dinner, galas, golf tournaments, and endurance events.
4. Planned Giving: Bequests, Charitable Remainder Unitrusts, Charitable Lead Trusts, Charitable Gift Annuities, IRA Rollovers, and other estate planning vehicles.

Distribution channels5 records

Marketing channels15 records

Core offering

Cure SMA is a 501(c)(3) nonprofit foundation that funds and coordinates Spinal Muscular Atrophy (SMA) research through academic and industry partners while providing direct support services to patients and families. Its core deliverables include research grant funding, an SMA Drug Pipeline tracker, a Care Center Network of clinical sites, the Annual SMA Conference, and educational publications and care resources for the SMA community, all provided free of charge.

Product overview

Cure SMA operates as a non-profit foundation offering a comprehensive portfolio of programs, publications, and resources to support the SMA community. The organization provides educational publications including the Directions community newsletter and Compass research publication, alongside clinical resources such as Care Series Booklets covering medical, breathing, and nutritional guidance. Their research infrastructure includes the SMA Drug Pipeline database and the Cure SMA Care Center Network. Community support is delivered through programs like the Annual SMA Conference, Walk-n-Roll fundraising events, and Summit of Strength local gatherings. The SMA Industry Collaboration initiative facilitates pharmaceutical partnerships to advance treatment development.

Differentiator

Problem solved

Functional benefit

Products and services

• Compass Publication Cure SMA's research publication covering scientific advances and clinical care developments in Spinal Muscular Atrophy, distributed to the SMA community.
• Directions Newsletter Community newsletter providing updates on SMA research, Cure SMA events, and community news, distributed to SMA families and supporters.
• Care Series Booklets Educational booklets providing guidance on medical issues, breathing care, musculoskeletal concerns, nutrition, and quality of life for individuals living with SMA.
• SMA Drug Pipeline Comprehensive resource tracking drug development programs for SMA treatments, currently covering 18+ programs in various stages of clinical development.
• Cure SMA Care Center Network Network of 10+ clinical research and treatment centers providing specialized SMA care, supporting a Clinical Data Registry and serving as a referral channel for newly diagnosed patients.
• Annual SMA Conference Annual in-person gathering bringing together SMA families, researchers, healthcare providers, and industry partners for education, networking, and community building; 900+ conference scholarships awarded to families annually.
• SMA Industry Collaboration Multi-phase collaborative initiative bringing together major pharmaceutical companies (Biogen, Novartis Gene Therapies, Genentech/Roche, Cytokinetics) to advance SMA research and treatment development.
• Annual SMA Research & Clinical Care Meeting Professional meeting held for 29+ years bringing together SMA scientists and clinicians worldwide, featuring 100+ research presentations and producing 16+ new research publications annually.
• Walk-n-Roll Community Fundraising Events Community fundraising events held across the United States to support SMA research, awareness, and Cure SMA programs.
• Summit of Strength Local events providing education, peer support, and networking opportunities for individuals and families affected by SMA across U.S. communities.
• Virtual SMA Webinars Online educational webinars covering SMA treatment updates, clinical trials, care guidelines, and wellness topics, delivered virtually to the SMA community.
• Virtual SMA Socials Virtual community gatherings designed for peer connection and social support among SMA community members regardless of geography.
• Cure SMA Research Grants Program Multi-million dollar annual research grant funding program supporting SMA investigators at universities and medical schools globally; awards typically range from $50,000 to $300,000 per project.

Quantifiable outcome

• Four FDA-approved SMA treatments developed with Cure SMA's involvement
• +3 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

Six partnerships are on record, tiered core, major and minor.

• Parent Project Muscular DystrophycoreStrategic or Co-development PartnerCure SMA and Parent Project Muscular Dystrophy announced a collaboration to jointly support the muscular dystrophy community, share resources, and coordinate advocacy efforts.
• SMA EuropemajorStrategic or Co-development PartnerCure SMA joined with SMA Europe and TREAT-NMD for a meeting with EMA regulators to discuss SMA therapies and regulatory pathways.
• TREAT-NMDmajorStrategic or Co-development PartnerInternational collaboration with TREAT-NMD for regulatory engagement with European Medicines Agency (EMA) on SMA treatments.
• The MightyminorStrategic or Co-development PartnerCure SMA partnered with The Mighty to share patient stories and expand reach of SMA awareness content.
• Muscular Dystrophy Association (MDA)majorStrategic or Co-development PartnerCure SMA announced partnership with MDA for newborn screening initiative, combining resources to advance early diagnosis of SMA.
• The District Policy GroupmajorStrategic or Co-development PartnerCure SMA announced partnership with The District Policy Group for federal advocacy efforts and legislative strategy.

Scale indicators8 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Parent Project Muscular Dystrophy (PPMD): Single-disease (Duchenne muscular dystrophy) nonprofit that Cure SMA explicitly collaborates with on muscular dystrophy community support and advocacy. Closest structural analog: a disease-specific foundation funding research, running conferences, and driving newborn screening.
• Muscular Dystrophy Association (MDA): Largest U.S. neuromuscular disease nonprofit, explicitly partnered with Cure SMA on newborn screening and shared neuromuscular research priorities. Comparable mission, revenue model (donations, events, corporate sponsorships), and Care Center/clinical network model.
• Huntington's Disease Society of America: Single-disease nonprofit funding HD research, supporting families, and running a Center of Excellence network. Comparable structure and revenue model; analogous in driving genetic-disease research and family-centered care.

Broad incumbents

• National Organization for Rare Disorders (NORD): U.S. umbrella rare disease advocacy organization representing 300+ disease-specific groups. Overlaps with Cure SMA on federal newborn screening advocacy and rare disease policy, though Cure SMA operates as a disease-specific group within NORD's ecosystem.
• ALS Association: Major U.S. neurodegenerative disease nonprofit funding research, running certified clinics, and advocating federally. Overlapping donor base and event-driven fundraising model with Cure SMA, though broader disease scope.
• Cystic Fibrosis Foundation: The archetype single-disease research nonprofit: funds basic and translational research, runs a care center network, and drove multiple FDA-approved CFTR modulators. Comparable operating model and scale; Cure SMA's SMA Industry Collaboration mirrors CFF's historic venture philanthropy model.

Emerging players

• Charcot-Marie-Tooth Association: Patient-driven nonprofit for CMT, another inherited neuromuscular disease with similar research-funding and patient-support model. Smaller scale than Cure SMA but same disease-category positioning and advocacy approach.

Others

• TREAT-NMD: Global neuromuscular disease research network that Cure SMA actively partners with on regulatory advocacy. Comparable in supporting clinical trial readiness and registries, but operates as an academic network rather than a donor-funded foundation.

Regional players

• Spinal Muscular Atrophy UK (SMA UK): U.K.-based SMA-specific charity that shares Cure SMA's disease mission and patient-support orientation but operates in a different geography. Comparable in scope and program mix (research funding, family services, advocacy) but U.K.-focused.
• SMA Europe: European SMA umbrella that Cure SMA formally partners with for EMA regulatory engagement. Comparable disease-specific mission and advocacy/research orientation, but Europe-focused; serves as Cure SMA's international counterpart.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/FamiliesofSMA
• YouTubeyoutube.com/channel/UCECjEl49gy32nz8wWeb2AFQ
• Facebookfacebook.com/familiesofsma
• LinkedInlinkedin.com/company/families-of-sma
• Instagraminstagram.com/curesmaorg
• TikToktiktok.com/@curesmaorg

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Spinal Muscular Atrophy Foundation