Resources

ENMC Poster Newborn Screening Alliance Congress Budapest.pdf

292nd ENMC workshop: Best practices after positive Spinal Muscular Atrophy newborn screening

2026 Congress_Research Priorities_Poster for advocates.pdf

Congress Research Priorities Poster for advocates - From Questions to Impact: Community-Driven Research Priorities in SMA

2026 Congress_Research Priorities_poster_final.pdf

Congress Research Priorities poster: From Questions to Impact: Community-Driven Research Priorities in SMA

259_EUPESMA25.pdf

European Patient Experience Survey in SMA (EUPESMA 2025): Treatment access, perceptions, and future needs

257_EUPESMA2025_ventilation_V2.pdf

Evolving phenotypes and ventilation needs in SMA in the post-treatment era: Results from a European community survey

260_EUPESMA23.pdf

Experiences of Clinical Trial Participation in SMA: Insights from a Pan-European SMA Europe survey

2026Congress_Medical emergencies_poster_final.pdf

Medical Emergencies in SMA: 1 st International Clinical Care Symposium

261_OdySMA SMA Europe poster congress Budapest_2026.pdf

OdySMA – Tracking access to SMA treatment and care through quantitative and qualitative data

2026_ECRD_Poster_DIGITAL_14MAY.pdf.pdf

Patients in the lead: embedding patient-led processes across advocacy domains at SMA Europe

256_Daily Life Study_Final.pdf

Real-time digital measurement of patient-experienced outcomes: An intensive longitudinal study in adults living with spinal muscular atrophy

263_System level barriers_ reimbursement gaps_ crowdfunding Member Survey SMA Europe poster congress Budapest_2026.pdf

System-level barriers, reimbursement gaps, and crowdfunding: insights from the SMA Europe Member Survey

262_The OdySMA Nations League_Descriptive benchmarking of system level access to therapies and care across Europe SMA Europe poster congress Budapest_2026.pdf

The OdySMA Nations League: Descriptive benchmarking of system-level access to therapies and care across Europe

255_EUPESMA25_Untreated - V2.pdf

When treatment either stops or never starts: Characteristics and experiences of people living with SMA in Europe

Scientific Posters by SMA Europe

Abstract Book - Barcelona 2022.pdf

Abstract Book. 3rd Scientific International Congress on Spinal Muscular Atrophy

Abstract Book Ghent 2024.pdf.pdf

Abstract Book. 4th Scientific International Congress on Spinal Muscular Atrophy

Congress26_poster and talk abstracts info_v2.pdf

Abstract Submission Guidelines for Posters and Oral Presentations

Policy on Animal Research 6b403c21b5.pdf

Animal research policy

SMA Europe is dedicated to improving the lives of people living with SMA and improve outcomes through high quality research to better understand and treat this condition.

Financial and Auditor's Report 2022.pdf

Audit Report 2022

Audit-report-2020-englisch.pdf

Audit report 2020

SMA-Europe e.V., Freiburg im Breisgau,&nbsp;Auditor's Report on the Fiscal Year 2020

2021 Financial Statement and Auditor's Report.pdf

Audit report 2021

CHMP opinion_Itvisma_24Apr26_final.pdf

Community update: Novartis receives EMA CHMP positive opinion for Itvisma

257_EUPESMA2025_ventilation_FINAL.pdf

Gewinnermittlg2020-2.pdf

Gewinnermittlg 2020

EINNAHMEN-AUSGABEN-&Uuml;BERSCHUSSRECHNUNG vom 01.01.2020 bis 31.12.2020

GSE_Posters_Display.pdf

Global SMAdvocacy Event 2026 Posters display

2026_Global SMAdvocacy event_Registration.pdf

Global SMAdvocacy Event 2026 Registration process

SMA Europe Membership Application Form.docx

Membership Application Form

SMA_Europe_List_of_Members_for_website.pdf

SMA Europe - List of Member Organisations

Creating a better world for all those living with SMA

SMA Europe Annual Report 2024.pdf

SMA Europe Annual Report 2023

SMA_InformeAnual_2025_final.pdf

SMA Europe Annual Report 2024

SMA-Europe-Code-of-Practice-on-Relationships-with-the-Pharmaceutical-Industry-Apr-16.pdf

SMA Europe Code of Practice on Relationships with the Pharmaceutical Industry

This Code of Good Practice is designed to guide relations between SMA Europe and the pharmaceutical industry (including their representatives and consultants)

EN_Financial Report 2022.pdf

SMA Europe Financial Report 2022

SMA_Europe_Tax_Exemption_Notice_2015-2017.pdf

SMA Europe Tax Exemption Notice 2015-2017

SMA_Europe_Tax_Exemption_Notice_2018.pdf

SMA Europe Tax Exemption Notice 2018

SMA_Europe_Tax_Exemption_Notice_2022.pdf

SMA Europe Tax Exemption Notice 2022

SMA_Europe_Tax_Exemption_Notice_2023.pdf

SMA Europe Tax Exemption Notice 2023

SMA_Europe_Tax_Exemption_Notice_2024.pdf

SMA Europe Tax Exemption Notice 2024

Info Sheet - SMA Patient Advocate Training_28-Apr26 2.pdf

SMA Patient Advocate Training Info Sheet

SMA Europe_Statutes_Dec_2022.pdf

Statutes of SMA Europe

SMA Europe official documents

DEVOTE WMS  Community Statement - SMA Europe - Oct 8 2024.pdf

Biogen Community Statement: results from Part B and C of the Phase 2/3 DEVOTE study

CT workshop paper - jnd_221538.pdf

Clinical trial readiness for spinal muscular atrophy: experience of an international educational-training initiative

risdiplam tablet EC Approval Community Letter_June 2025.pdf

Community Update Roche: EMA approval of the risdiplam tablet

End of Year SMA Community Letter 2024.pdf

Community Update Roche: End of Year Community Letter

Community Letter_March 2024 Congresses.pdf

Community letter Roche. March 2024

RESILIENT Letter to SMA Community Dec2024-AN.pdf

Community update Biohaven: Biohaven's RESILIENT Letter to SMA Community

Community_Letter_March_2024_Congresses_27 March 2024.pdf

Community update Roche: Findings on the risdiplam tablet formulation and next steps

Roche_risdiplam_CHMP_recommendation_Community_letter_FINAL_21 July 2023.pdf

Community update Roche: Risdiplam_CHMP_recommendation

Roche_risdiplam RAINBOWFISH Two-Year Update_WMS 2024.pdf

Community update Roche: Risdiplam_Rainbowfish_two years update

DEVOTE Part B Top Line  Community Statement - SMA Europe - Sept 4 2024.pdf

DEVOTE Part B Top Line  Community Statement: Biogen

EVRY2109 Patient lay summary_FIREFISH_v6.pdf

FIREFISH Patient Summary

An updated summary of long-term (2-year) data from FIREFISH, a clinical trial to establish the efficacy and safety of risdiplam for children with Type 1 SMA

CT Workshop - Evry 2020.pdf

First clinical trials best practice workshop

As part of its mission to bring effective therapies to patients in a timely &amp; sustainable way, we have started to expand Europe&rsquo;s current capacity to run clinical trials for potential SMA therapies.

41591_2024_Article_3484 (3) 2.pdf

First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy

Infopack-for-Clinical-research-in-condusting-Clinical-trials-for-SMA_Part-1_SMA_Pathology-diagnosis-clinical-presentation-therapeutic-strategies-and-treatments.pdf

Infopack for Clinical research in conducting Clinical trials for SMA Part-1

Pathology, diagnosis, clinical presentation, therapeutic strategies &amp; treatments

SMA_Lay Patient Summary_JEWELFISH 1Y_2021.pdf

JEWELFISH Patient Summary

A summary of 1-year data from JEWELFISH, a clinical trial to establish the safety, tolerability and pharmacokinetics/pharmacodynamics of risdiplam in individuals with SMA who were previously treated with other SMA therapies

SMA_FIREFISH_Lay-person-summary_Updated-version.pdf

SMA FIREFISH Lay person summary

A clinical trial to establish the efficacy and safety of risdiplam for babies with Type 1 SMA (FIREFISH)

SMA_SUNFISH_Lay-person-summary_Updated-version.pdf

SMA SUNFISH Lay person summary

A clinical trial to establish the efficacy and safety of risdiplam for children and adults with SMA Types 2 and 3 (SUNFISH)

EVRY2109 Patient lay summary_SUNFISH_v5.pdf

SUNFISH Patient Summary

An updated summary of long-term (2-year) data from SUNFISH, a clinical trial to establish the efficacy and safety of risdiplam for children, adolescents and adults with Type 2 or 3 SMA

Scholar-Rock-Announces-Positive-Interim-Results-from-Phase-1-Trial-of-SRK-015-in-Healthy-Volunteers-and-Updates-on-Future-Development-Plans.pdf

Scholar Rock Announces Positive Interim Results from Phase 1 Trial

Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

SCHOLA~3.PDF

Scholar Rock Reports Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE

Scholar-Rock-Reports-First-Quarter-2020-Financial-Results-and-Highlights-Business-Progress.pdf

Scholar Rock Reports First Quarter 2020 Financial Results and Highlights Business Progress

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the first quarter ended March 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs.

Scholar Rock SAPPHIRE Community Statement_Final.pdf

Scholar Rock SAPPHIRE Community Statement October 2024

Spinraza-Adult-Consumer-Brochure.pdf

Spinraza Adult Consumer Brochure

For the more than 3200 adults who have been treated with SPINRAZA worldwide, victories are personal.

Spinraza-Pediatric-Consumer-Brochure.pdf

Spinraza Pediatric Consumer Brochure

For the more than 10,000 people who have been treated with SPINRAZA worldwide, victories are personal.

DEVOTE Study Brochure.pdf

The DEVOTE Study

The DEVOTE Study is a clinical trial initiated by Biogen that will evaluate the safety and efficacy of higher doses of nusinersen

Nurture Study Brochure_29.09.21.pdf

The NURTURE Study

A clinical study in newborn babies with a genetic diagnosis of Spinal Muscular Atrophy (SMA)

SMA Respond Study Patient Advocacy Groups Brochure 2021 ECP .pdf

The RESPOND Study

A clinical study in young children with Spinal Muscular Atrophy (SMA)

US FDA Approval_OAV101 Community Statement SMA Europe.pdf

US FDA Approval of OAV101 IT

Treated_with_Risdiplam.pdf

When treated with Risdiplam

Like nusinersen, risdiplam is a selective SMN2 gene splicing modifier.

Treated_with_ASO.pdf

When treated with an ASO

ASOs are small snippets of synthetic genetic material that bind ribonucleic acid (RNA).

Treatments & clinical trials

Advances 2024 in SMN1-related proximal SMA_AFM Telethon.pdf

AFM Télethon publication: Advances 2024 in SMN1-related proximal spinal muscular atrophy

NBS-Dangouloff-ENMC-2019.pdf

NBS Dangouloff ENMC 2019

244th ENMC international workshop: Newborn screening in spinal muscular atrophy, May 10&ndash;12, 2019, Hoofdorp, The Netherlands

Biogen-Patient-Brochures_Navigating-health-information-and-services-_2021-05-27_FINAL.pdf

Navigating health information and services

When it comes to your health, playing an active role in decision making can bring many benefits.

SMA_White-Paper-NBSA_v8.pdf

Spinal Muscular Atrophy - Screen at birth, save lives - White Paper 2024

Infopack 2 - Standards of Care - Clean copy - March 22.pdf

Standards of Care for SMA

Biogen-Patient-Brochures_Understanding-scientific-and-clinical-developments-_2021-05-27_FINAL.pdf

Understanding scientific and clinical developments

As new potential medicines are studied in clinical trials, you may be interested to understand more about the research and regulatory process which ensures that new medicines work well for patients and do not cause unacceptable side effects.

Understanding-the-MFM-the-SMAIS-in-the-Context-of-Outcome-Measurements-in-SMA-Final.pdf

Understanding the MFM & the SMAIS in the Context of Outcome Measurements in SMA

This brochure was created to provide additional information about the Motor Function Measure (MFM-32) and the SMA Independence Scale (SMAIS) in the context of other outcome measures used in SMA.&nbsp;

Living with SMA: useful documents

Congress 2022 A decision for life.pdf

A Decision for Life – pathway to treatment decisions in newly diagnosed families with SMA. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

ABN_Neurology_COVID-19_Guidance_22.3.20.pdf

ABN Neurology COVID-19 Guidance

The ABN recognises that this is a rapidly changing field. We will update our advice as we learn more.

The need for evidence-based treatment decisions in spinal muscular atrophy. Annals of Translational Neurology..pdf

Annals of Translational Neurology. "The need for evidence-based treatment decisions in spinal muscular atrophy."

CT Site Readiness - Poster for Orlando - June 23.pdf

Clinical Trials Readiness in Europe. Scientific Poster. Cure SMA Conference. Orlando 2023.

CT Workshop Poster - Orlando 2023.pdf

Clinical Trials Workshop Scientific Poster. Cure SMA Conference. Orlando 2023.

Disease Burden & Patient Treatment ExpectationsNMD 2017.pdf

Disease impact on general well-being and therapeutic expectations of European type 2 and 3 spinal muscular atrophy patients. The first pan-European survey.

EUPESMA-2019_Lay summary_v4.pdf

EUPESMA - 2019 Patient Summary

A summary of the EUPESMA-2019 survey results

Congress 2022 EUPESMA 2019.pdf

EUPESMA 2019 - European Patient Experience Survey in SMA. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

Congress 2022 poster EUPESMA 2021.pdf

EUPESMA 2021 – European Patient Expectation Survey in SMA. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

EUPESMA Launch Flyer - English.pdf

EUPESMA 2025 Flyer

SMA Europe Publications and Reports

Resources

Scientific Posters by SMA Europe (13)

SMA Europe official documents (25)

Treatments & clinical trials (30)

Living with SMA: useful documents (7)

SMA Europe Publications and Reports (34)

Community updates (48)

Comms & marketing (40)

Other (7)

Categories

Scientific Posters by SMA Europe (13)

SMA Europe official documents (25)SizeFormatUpdated

SMA Europe official documents (25)

Treatments & clinical trials (30)SizeFormatUpdated

Treatments & clinical trials (30)

Living with SMA: useful documents (7)SizeFormatUpdated

Living with SMA: useful documents (7)

SMA Europe Publications and Reports (34)SizeFormatUpdated

SMA Europe Publications and Reports (34)

Community updates (48)SizeFormatUpdated

Community updates (48)

Comms & marketing (40)SizeFormatUpdated

Comms & marketing (40)

Other (7)SizeFormatUpdated

Other (7)

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