Resources

ENMC Poster Newborn Screening Alliance Congress Budapest.pdf

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

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.

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

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.

259_EUPESMA25.pdf

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

257_EUPESMA2025_ventilation_FINAL.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

Congress 2022 Fatigue, Pain, & Psychological problems.pdf

Impact of Fatigue, Pain and Psychological Problems on Perceived Quality of Life in Paediatric SMA Patients. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

2026Congress_Medical emergencies_poster_final.pdf

Medical Emergencies in SMA: 1 st International Clinical Care Symposium

TAMAÑO A0_ 221006_SMA Europe poster congress OdySMA_2022_final_A0.pdf

OdySMA - SMA Europe Advocacy Tool. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

230530_SMA Europe poster CureSMA congress OdySMA_2023_ng_lg.pdf

OdySMA 2023. Scientific Poster. Cure SMA Conference. Orlando 2023.

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

PSP Poster for Orlando -  23 May 2023.pdf

Priority Setting Project. Scientific Poster. Cure SMA Conference. Orlando 2023.

S95_MDA2024_RESTORE_Vent_Support_FINAL.pdf

Real-World Outcomes Following Onasemnogene Abeparvovec  in Patients with Spinal Muscular Atrophy and Invasive  Ventilatory Support: Findings from the RESTORE Registry

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

Congress 2022 Daily Life Study.pdf

The SMA Daily Life Study. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

CureSMA2023_Daily Life Study_FINAL.pdf

The SMA Daily Life Study. Scientific Poster. Cure SMA Conference. Orlando 2023.

Congress 2022 Type 0.pdf

Type 0. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

Ukrainian SMA registry. Scientific Poster. 3rd Scientific Congress on SMA. Barcelona 2022.

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

13Call_Research_2026_Eligibility&Procedure_final2.pdf

Call for Research 13 Eligibility and Procedure

Gewinnermittlg2020-2.pdf

Gewinnermittlg 2020

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

Leaflet Global SMAdvocacy Event 2026.pdf

Global SMAdvocacy Event 2026 Guide

GSE_Posters_Display.pdf

Global SMAdvocacy Event 2026 Posters display

2026_Global SMAdvocacy event_Registration.pdf

Global SMAdvocacy Event 2026 Registration process

Final_Global SMAdvocacy Event Report.pdf

Global SMAdvocacy Event Activity Report

SMA Europe Membership Application Form.docx

Membership Application Form

Final_Application Form_SMA Patient Advocate Training.pdf

Patient Advocate Training

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_Annual_Report_2024_Full_5bdaf12189.pdf

SMA Europe Annual report 2025.pdf

SMA Europe Annual Report 2025

SMA Europe Annual Report 2026

SMA_Europe_Code_of_Practice_on_Relationships_with_the_Pharmaceutical_Industry_Apr_16_5e8d881079.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

SMAEurope_Symposium25_Abstract info_May25.pdf

SMA Europe Symposium 2025 Abstract Requirements

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 & reports 

CT workshop paper - jnd_221538.pdf

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

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.

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).

Treatment & Clinical Trial resources

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: guides & resources

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.

Publications 

Resources

Scientific Posters by SMA Europe (26)

SMA Europe official documents & reports (31)

Treatment & Clinical Trial resources (21)

Living with SMA: guides & resources (7)

Publications (18)

Community Letters & Updates (59)

Communication & Marketing Resources (43)

Other resources and updates (4)

Categories

Scientific Posters by SMA Europe (26)

SMA Europe official documents & reports (31)SizeFormatUpdated

SMA Europe official documents & reports (31)

Treatment & Clinical Trial resources (21)SizeFormatUpdated

Treatment & Clinical Trial resources (21)

Living with SMA: guides & resources (7)SizeFormatUpdated

Living with SMA: guides & resources (7)

Publications (18)SizeFormatUpdated

Publications (18)

Community Letters & Updates (59)SizeFormatUpdated

Community Letters & Updates (59)

Communication & Marketing Resources (43)SizeFormatUpdated

Communication & Marketing Resources (43)

Other resources and updates (4)SizeFormatUpdated

Other resources and updates (4)

Categories