Resources

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.

PSP Poster for Orlando -  23 May 2023.pdf

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

Congress 2022 Daily Life Study.pdf

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

Scientific Posters by SMA Europe

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

DEVOTE Part B Top Line  Community Statement: Biogen

41591_2024_Article_3484 (3) 2.pdf

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

SCHOLA~3.PDF

Scholar Rock Reports Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE

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.

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

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.

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.

Advances_SMA.pdf

Advances in research 2026 Spinal muscular atrophy (SMA)

A Decision for Life e Treatment decisions in newly diagnosed families. European Journal of Paediatric Neurology..pdf

European Journal of Paediatric Neurology. "A Decision for Life - Treatment decisions in newly diagnosed families."

s13023-026-04588-9_reference.pdf

From questions to impact in spinal muscular atrophy - identifying community-driven research priorities through a multi-stakeholder European initiative

Publications 

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

Community Statement_BIOCAD.pdf

Community Statement BIOCAD July 2023

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

SMA Community Statement Cure SMA 2023 FINAL.pdf

Community Update from Biogen after Cure SMA 2023 Conference

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

MDA STEER STRENGTH Community Statement_Mar25.pdf

Community update Novartis: Phase III STEER and Phase IIIb STRENGTH studies of OAV101 IT

MDA 2025 SUNFISH Community Letter_Mar25.pdf

Community update Roche: Final five-year data from the pivotal SUNFISH

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

CHMP opinion_Itvisma_24Apr26_final.pdf

Community update: Novartis receives EMA CHMP positive opinion for Itvisma

EMEA-SMART-Community-Statement-April-2021-FINAL-_440418037.pdf

EMEA SMART Community Statement

Community Statement from Novartis Gene Therapies

Roche End of Year (2023) SMA Community Letter.docx - Google Docs.pdf

End of Year 2023: Roche's Community Letter

ScholarRock_BLAAcceptance_CommunityLetter_5.7.26.pdf

FDA accepts to review Biologics License Application for apitegromab

EMEA_GTx Community Update JUNE.pdf

Novartis Gene Therapies Community Update June 2022

Novartis-Global-Media-Release-AVXS-101-IT-Clinical-Hold-FINAL-October_30_2019_7am_CET-1.pdf

Novartis Global Media Release

Novartis announces AVXS-101 intrathecal study update

Roche-Community-Letter-CHMP-Opinion.pdf

Roche Community Letter CHMP Opinion

As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development programme, we are delighted to share an important milestone on our European regulatory submission.

Roche-Community-Letter-FDA-File-Acceptance-Nov.-2019.pdf

Roche Community Letter FDA File Acceptance

As part of our ongoing partnership and following your request to receive timely information about the risdiplam clinical development program, we are pleased to share with you that the U.S. Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for risdiplam for the proposed use in people living with Spinal Muscular Atrophy (SMA).

Roche_Community_Letter-Risdiplam_EC_Approval.pdf

Roche Community Letter Risdiplam EC Approval

We are delighted to be able to share with you &ndash; as per your request for regular updates &ndash; that today the European Commission (EC) has approved Evrysdi&trade; (risdiplam), the first and only at home Spinal Muscular Atrophy (SMA) treatment with proven efficacy in adults, children and babies two months and older.

Roche-Community-Letter-Risdiplam-FDA-approval-SMA-Europe.pdf

Roche Community Letter Risdiplam FDA approval SMA Europe

As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we are delighted to share with you a much-anticipated milestone.

Roche-Cure-SMA-Conference-Community-letter.pdf

Roche Cure SMA Conference Community letter

We are pleased to share new interim data from two studies evaluating risdiplam in people with spinal muscular atrophy (SMA) who have been previously treated with other SMA-targeting therapies (JEWELFISH) and pre-symptomatic babies (RAINBOWFISH).&nbsp;

Roche_End_of_Year_Community_Letter-SMA-EU.pdf

Roche End of Year Community Letter SMA EU

As 2020 draws to a close, based on your requests to receive regular updates, we would like to provide you with a summary of our work in the community throughout this year, including information about the risdiplam clinical development programme and progress towards our goal of making risdiplam available to all patients who can benefit from the treatment

Roche-Regulatory-Update-Community-Letter-SMA-Europe.pdf

Roche Regulatory Update Community Letter SMA Europe

As part of our ongoing partnership and following your request to receive important information about the risdiplam clinical development program, we wanted to update you on the status of regulatory filings and reviews of risdiplam around the world.

Roche_Year_in_-Summary_Community-Letter_SMA-Europe.pdf

Roche Year in Summary Community Letter SMA Europe

As we come to the close of 2021, based on your requests to receive regular updates, we would like to provide an overview of our recent work in the community over the past year and a summary of our progress to advancing research and increasing access to risdiplam in Europe and beyond.

Roche-community-update-MAA-Validation-SMA-Europe.pdf

Roche community update MAA Validation SMA Europe

We are pleased to be in touch again so soon following the positive FDA announcement last week - this time to share an important milestone on our European regulatory submission.

roche-media-release-risdiplam-28042020.pdf

Roche media release risdiplam

Roche&rsquo;s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)

SMA-EU-Community-Update-090919.docx

SMA EU Community Update

In response to your request for an update, please find an update on access to SPINRAZA (nusinersen) in Europe.

Scholar Rock Update for SMA Europe.SMA Treatment FDA Approval.pdf

Scholar Rock Community Letter: FDA approval 11 September 2026

Community Letters & Updates

Resources

Scientific Posters by SMA Europe (3)

Treatment & Clinical Trial resources (7)

Living with SMA: guides & resources (4)

Publications (4)

Community Letters & Updates (29)

Categories

Scientific Posters by SMA Europe (3)

Treatment & Clinical Trial resources (7)SizeFormatUpdated

Treatment & Clinical Trial resources (7)

Living with SMA: guides & resources (4)SizeFormatUpdated

Living with SMA: guides & resources (4)

Publications (4)SizeFormatUpdated

Publications (4)

Community Letters & Updates (29)SizeFormatUpdated

Community Letters & Updates (29)

Categories