Fundacja SMA

What Fundacja SMA does

Fundacja SMA is a Polish non-profit patient advocacy foundation established in 2013 to support individuals affected by Spinal Muscular Atrophy (SMA), a rare genetic neuromuscular disease. The foundation serves approximately 1,000 SMA patients in Poland and is led by volunteers who are themselves parents of children with SMA, lending the organization direct credibility with its beneficiary population. Its core activities span treatment access advocacy, patient and family support services, and coordination of clinical-care infrastructure across the Polish healthcare system.

The foundation's core products and service infrastructure include: (i) coordination of a 36-hospital drug program delivering NFZ-reimbursed SMA therapies (Spinraza, Zolgensma, and Evrysdi); (ii) maintenance of a national SMA patient registry; (iii) a medical equipment lending bank for patients and families; (iv) the ACTIVE-NBS newborn screening pilot project; and (v) annual community programming such as the Weekend ze SMA-kiem conference. Since 2014 it has been a member of SMA Europe, EURORDIS, and TREAT-NMD, giving it international advocacy reach. The organization played a central role in securing NFZ reimbursement for all three currently approved SMA drugs, making Poland one of the more comprehensive-access markets for SMA therapy.

The foundation operates a non-profit funding model with no commercial revenue. Income is derived from individual donations, the Polish 1.5% personal-income-tax earmark program, and institutional grants — primarily from PFRON (State Fund for Rehabilitation of Disabled Persons), co-financing from MRiPS (Ministry of Family and Social Policy), and international rare-disease organizations. It has no disclosed headcount beyond volunteer leadership and no publicly audited financials. There are no products sold, no pricing model, and no commercial customer segments; the foundation's "customers" in a strategic sense are SMA patients and their families, served free of charge.

Name

Fundacja SMA

Legal name

Fundacja SMA

Website

https://fsma.pl

Company type

Private

Founded year

2013

Operating status

Operating

Headcount range

1–10 employees

Short description

Fundacja SMA is a Polish non-profit foundation established in 2013 that advocates for and supports approximately 1,000 patients with Spinal Muscular Atrophy through treatment-access coordination, a patient registry, equipment lending, and family programming.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy and Support Services

NAICS

Voluntary Health Organizations (813212), Social Advocacy Organizations (8133), Grantmaking Foundations (813211)

SIC

Services-Misc Health & Allied Services, Nec (8090)

akta.pro primary industry

Public Health Financing, Budgeting & Grants Management (HLAJAMAC)

Keywords

• Patient advocacy services
• Rare disease support
• Nonprofit foundation services
• Healthcare community education
• Patient registry operations

Headquarters

HQ city

Warsaw

HQ country

Poland

HQ region

Europe

Markets served

GTM type

B2C

Offering type

Services

Cost components

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

Revenue model

1. Donations and charitable contributions: Fundacja SMA raises funds through individual donations, corporate sponsorships, charity events (SMArt Run, Podniebna Drużyna parachute events), and the Polish 1.5% personal income tax donation program (przekaż 1.5% podatku). These funds support the foundation's operations, patient support programs, equipment bank (Bank Sprzętu), and advocacy activities.

Go-to-market motion2 records

Distribution channels4 records

Marketing channels10 records

Core offering

Fundacja SMA is a Polish patient advocacy foundation that provides free education, peer support, advocacy, and equipment loans to people living with Spinal Muscular Atrophy (SMA) and their families. Its core offerings include an annual SMA conference, an educational webinar series, printed care publications, an equipment loan bank, an online patient database and registry, and community-building events such as charity runs and parachute jumps. The foundation's secondary role is healthcare-system advocacy — it played a decisive role in securing NFZ reimbursement for Spinraza, Zolgensma, and Evrysdi and in introducing nationwide newborn screening for SMA in Poland.

Product overview

Fundacja SMA is a Polish patient advocacy foundation for Spinal Muscular Atrophy (SMA). Its primary product is the comprehensive informational website fsma.pl, which serves as a central hub providing disease education, treatment guidance, a patient registry (Polska Baza SMA), and community support resources. The foundation also produces educational publications including the annual 'Bilans SMA'KA' report and practical care guides such as 'Z SMA1 na co dzień.' Educational services include a webinar series (Webinary) and the annual 'Weekend ze SMA-kiem' conference. The foundation supports access to three reimbursed SMA therapies in Poland (Spinraza/nusinersen, Zolgensma/gene therapy, and Evrysdi/risdiplam) through advocacy and the national treatment program B.102.FM. It also tracks experimental therapies including Reldesemtiv, Apitegromab, RO7204239, Taldefgrobep alfa, BIIB115/Salanersen, and ANB-004. Community initiatives include 'Drużyna SMA' (running community), 'Handmade for Hope' (fundraising), and research projects such as 'Aktywnie z SMA' (rehabilitation) and 'ACTIVE-NBS' (motor development research). The organization is a member of SMA Europe and EURORDIS, operating under the slogan 'Razem pokonamy rdzeniowy zanik mięśni' (Together we will overcome spinal muscular atrophy).

Differentiator

Problem solved

Functional benefit

Brands

• Weekend ze SMA-kiem: Doroczna konferencja dla osób żyjących z rdzeniowym zanikiem mięśni, ich rodzin, opiekunów oraz specjalistów. Najważniejsze w Polsce spotkanie społeczności SMA.
• SMArt Run
• Drużyna SMA
• Podniebna Drużyna SMA
• Handmade for Hope
• Polska Baza SMA
• Bilans SMA'KA

Products and services

• Informational Web Platform (fsma.pl) Central web portal providing comprehensive information about Spinal Muscular Atrophy, including disease education, treatment guidance, patient registry access, educational publications, webinar access, and community support for SMA patients and families in Poland.
• Annual Conference 'Weekend ze SMA-kiem' Annual conference (XIV edition held in June 2026 in Warsaw) bringing together SMA patients, families, caregivers, and specialists to discuss new therapies, treatment accessibility, multidisciplinary care, and rehabilitation. Held under the patronage of the Chancellery of the President of Poland and attended by 113 families in 2026.
• Educational Webinar Series Online educational webinar series covering SMA-relevant topics including diet, spinal health, neurological speech therapy, PNF physiotherapy, massage, respiratory physiotherapy, orthopedic equipment, and therapy for newly diagnosed families, featuring expert presenters from Poland and abroad.
• Bilans SMA'KA Publication Annual informational publication by Fundacja SMA covering the state of SMA care, treatment updates, and practical guidance for patients and families. Latest edition published July 2026.
• SMA Vademecum: Z SMA1 na co dzień Practical care guide for families of children with the most severe form of SMA (Type 1), providing day-to-day care guidance and treatment support.
• Polska Baza SMA (Polish SMA Database) Online patient database operated by Fundacja SMA to monitor the state of SMA treatment in Poland and facilitate contact between the foundation and patients.
• Polski Rejestr Pacjentów SMA (Polish SMA Patient Registry) Patient registry established in January 2010 under TREAT-NMD, managed by the Neurology Department of Warsaw Medical University, supporting clinical trial participation and international collaboration.
• Handmade for Hope Fundraising Campaign Fundraising campaign producing and selling handmade items to raise funds for SMA research and patient support at Fundacja SMA.
• Drużyna SMA Running Community Running-based community initiative where participants dedicate their athletic efforts to SMA patients, with annual kilometer collection campaigns supporting the foundation.
• ACTIVE-NBS Research Project International research project 'Liczy się każdy kroczek' studying gross motor development in children diagnosed with SMA through newborn screening, using sensor-equipped suits and smart ankle bands to monitor motor function.
• Równe możliwości Support Project Support project for SMA families from Ukraine seeking refuge in Poland from the war, providing funding for genetic testing, medical consultations, psychotherapy, orthopedic equipment, accommodation, and participation in foundation events.
• Mój Asystent 2023 Personal Assistant Program Program 'Asystent osobisty osoby z niepełnosprawnościami – edycja 2023' providing 660 hours of personal assistant support (averaging 60 hours per month) to 24 adult SMA patients, funded by the Ministry of Family and Social Policy.
• Aktywnie z SMA IV Rehabilitation Project Comprehensive rehabilitation project co-funded by PFRON (1,709,930 PLN), providing 66 rehabilitation sessions per participant, personal assistant services (averaging 330 hours), and personal development training for 90 SMA participants.

Quantifiable outcome

• All three SMA drugs (Spinraza, Zolgensma, Evrysdi) are now reimbursed by NFZ for all eligible patients in Poland
• +7 more outcomes

Named customers2 records

Segments4 records

Ideal customer profiles4 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

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

• SMA EuropecoreStrategic or Co-development Partner · 1 March 2014Fundacja SMA has been a full member of SMA Europe since March 2014, participating in regular meetings and co-deciding on the direction of this international association. SMA Europe funds research programs and has collectively raised over €5 million for SMA research over the past 10 years. Fundacja SMA has been contributing to SMA Europe's grant fund since 2015.
• EURORDIS (European Organisation for Rare Diseases)coreStrategic or Co-development Partner · 1 January 2014Since 2014, Fundacja SMA has been a member of EURORDIS, which supports coordination between patient organizations, pharmaceutical industry, and health institutions, organizes training for patient organizations, and advocates for improved access to treatments for rare diseases in Europe.
• TREAT-NMDcoreStrategic or Co-development Partner · 1 January 2014Since 2014, Fundacja SMA has been a member of TREAT-NMD, a pan-European network for neuromuscular diseases. The Polish SMA Registry operates under the auspices of TREAT-NMD and is managed by the Faculty of Neurology at Warsaw Medical University.
• RochemajorStrategic or Co-development PartnerRoche co-developed risdiplam (Evrysdi) with PTC Therapeutics and SMA Foundation. Fundacja SMA actively facilitated the inclusion of Polish patients in all four risdiplam clinical trials (FIREFISH, SUNFISH, RAINBOWFISH, JEWELFISH), with Poland having the highest number of children in SUNFISH (33) and Polish infants representing 1/4 of all participants in FIREFISH.
• PTC TherapeuticsmajorStrategic or Co-development PartnerPTC Therapeutics co-developed risdiplam (Evrysdi) with Roche and SMA Foundation.
• SMA Foundation (USA)majorStrategic or Co-development PartnerSMA Foundation funded the discovery of risdiplam and the majority of its further development. Founded by the father of a girl with SMA. Fundacja SMA has partnered with SMA Foundation Russia to jointly enable 70% of FIREFISH trial participants, accelerating the development of risdiplam by at least one year.
• Novartis Gene Therapies (formerly AveXis)majorStrategic or Co-development PartnerFundacja SMA, acting within SMA Europe, supports the work of Novartis Gene Therapies — manufacturer of Zolgensma — in developing and making gene therapy available in Europe. The foundation participated in developing the protocol for European clinical trials and collaborated with the European Medicines Agency on market authorization.
• BiogenmajorStrategic or Co-development PartnerBiogen developed and markets Spinraza (nusinersen), the first approved SMA treatment. Fundacja SMA's advocacy campaign was instrumental in securing NFZ reimbursement for Spinraza in Poland from January 2019. Biogen is also developing BIIB115/salanersen as a successor to Spinraza.
• AFM Téléthon (France)majorStrategic or Co-development PartnerAFM Téléthon funds the Paris Institut de Myologie, where Dr. Martine Barkats first proved that gene therapy could be used to treat SMA. The Institut de Myologie is entirely funded by AFM Téléthon. Fundacja SMA co-financed subsequent research. AFM Téléthon is also a co-organizer of the International SMA Congress (Evry 2020).
• CytokineticsminorStrategic or Co-development PartnerCytokinetics developed reldesemtiv, an experimental drug for improving muscle function in SMA patients. The research began in 2014 with a grant from CureSMA.
• Astellas (partner to Cytokinetics)minorStrategic or Co-development PartnerAstellas partnered with Cytokinetics to advance the development of reldesemtiv for SMA.
• Scholar RockminorStrategic or Co-development PartnerScholar Rock developed apitegromab (SRK-015), an experimental drug for supplementary treatment of SMA types 2 and 3. The drug received FDA Fast Track designation in May 2021. Fundacja SMA facilitated the TOPAZ clinical trial.
• Roche (for RO7204239)minorStrategic or Co-development PartnerRoche is developing RO7204239 (GYM329), a myostatin inhibitor for improving muscle function in SMA patients. The MANATEE Phase 2/3 clinical trial is being conducted in Poland at three sites: Gdańsk Medical University, Poznań Medical University, and Warsaw Medical University.
• Biohaven PharmaceuticalsminorStrategic or Co-development PartnerBiohaven Pharmaceuticals is developing taldefgrobep alfa (BHV-2000) for supplementary treatment of SMA. The Phase III RESILIENT clinical trial is being conducted in Poland at four sites: Gdańsk Medical University, Poznań Medical University, Centrum Zdrowia Dziecka in Warsaw, and Dolnośląski Szpital Specjalistyczny in Wrocław.
• Ionis Pharmaceuticals and Biogen (for BIIB115)minorStrategic or Co-development PartnerBIIB115 (ION306) is being co-developed by Ionis Pharmaceuticals and Biogen as a successor to Spinraza. It is administered intrathecally like Spinraza but aims to be more effective and require less frequent dosing (every 6-12 months). Preparations for further clinical trials in Poland are underway.
• Biocad (Russia) and University of Massachusetts (for ANB-004)minorStrategic or Co-development PartnerANB-004 is an experimental gene therapy for SMA being developed by Russian company Biocad in collaboration with the University of Massachusetts. Like Zolgensma, it uses an AAV9 virus vector. The BLUEBELL Phase 1/2 clinical trial began in February 2023 and is being conducted in Russia (Moscow, Saint Petersburg, Yekaterinburg).
• Warsaw Sharks (Polish American Football Team)minorGTM or Marketing PartnerFundacja SMA partnered with the Warsaw Sharks American football team for the 'Mięśnie dla Mięśni' (Muscles for Muscles) campaign in 2014, using sports events to raise awareness about SMA and collect donations for the equipment bank.

Scale indicators6 records

Recent moves5 records

Expansion highlights4 records

Regional players

• SMA Finland: Finland's SMA patient organization, co-funder of Fundacja SMA's Ukraine initiative. Regional peer with the same patient-advocacy operating model but a separate national constituency.

Broad incumbents

• Muscular Dystrophy Association (MDA): Large US neuromuscular umbrella organization covering SMA among many conditions. Broader incumbent with overlapping patient-support and research-funding activities but not specializing in SMA.

Direct peers

• TREAT-NMD: Global neuromuscular-disease research network operating the SMA Patient Registry under which Fundacja SMA's Polish registry is run. Comparable as a neuromuscular patient-data and clinical-trial infrastructure organization.
• TreatSMA: UK-based SMA charity co-funding advocacy projects with Fundacja SMA (e.g., Ukraine refugee support). Direct peer as a small national SMA patient-advocacy and research-funding organization.
• EURORDIS - Rare Diseases Europe: European federation for rare-disease patient organizations; Fundacja SMA is a member since 2014. Direct peer in policy advocacy, training and access-to-treatment lobbying across European rare-disease communities.
• AFM-Téléthon (France): French neuromuscular patient organization and major SMA/gene-therapy research funder (Institut de Myologie). Direct peer in patient advocacy plus disease-modifying research funding, and co-organizer of the International SMA Congress.
• Spinal Muscular Atrophy UK (SMA UK): UK SMA patient organization with the same mission of patient support, research funding, and treatment-access advocacy. Direct functional peer, providing joint co-funding to Fundacja SMA projects such as the Ukraine refugee initiative.
• Cure SMA (USA): Largest US SMA patient advocacy organization funding SMA research and supporting families. Direct peer in mission and operating model, and historical grant-maker for the Cytokinetics reldesemtiv program that Fundacja SMA also supports.
• SMA Europe: Umbrella federation of European SMA patient organizations of which Fundacja SMA is a full member since 2014. Directly comparable as a coordinating SMA patient-advocacy body, with overlapping governance, fundraising and research-funding roles.
• SMA Foundation (USA): US foundation that funded the discovery of risdiplam; partner to Fundacja SMA through the joint FIREFISH enrollment effort that accelerated risdiplam development by ~1 year. Direct peer in funding SMA drug discovery and clinical-trial enablement.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/fundacjasma
• YouTubeyoutube.com/fundacjasma
• Facebookfacebook.com/fundacjasma
• LinkedInlinkedin.com/company/fundacjasma
• Instagraminstagram.com/fundacjasma

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Fundacja SMA