Resources

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

Community Letter_March 2024 Congresses.pdf

Community letter Roche. March 2024

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.

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)

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

Abstract Book Ghent 2024.pdf

Abstract Book from the Scientific International Congress on SMA

Policy on animal research.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

Gewinnermittlg2020-2.pdf

Gewinnermittlg 2020

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

Membership Application Form

SMA_Europe_List_of_Members_February 2024.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-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_Statutes_Dec_2022.pdf

Statutes of SMA Europe

SMA Europe official documents

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.

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.

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."

Final_Global SMAdvocacy Event Report.pdf

Global SMAdvocacy Event Activity Report

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.

Impact of Fatigue, Pain, and Psychological Problems on Perceived Quality of Life in Pediatric SMA Patients. Thieme Connect..pdf

Neuropediatrics 2021. "Impact of Fatigue, Pain, and Psychological Problems on Perceived Quality of Life in Pediatric SMA Patients."

Nutrition in SMA meeting report_v4.0_20Jul23.pdf

Nutrition in SMA Workshop: report

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.

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

schroth-et-al-2024-SMA-update-in-best-practices_treatment_considerations_NCP (002).pdf

Spinal Muscular Atrophy Update in Best Practices Recommendations forTreatment Considerations

Systematic Literature Review of the Natural History of SMA.pdf

Systematic Literature Review of the Natural History of Spinal Muscular Atrophy

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.

The diagnosis communication process in SMA_paper.pdf

The diagnosis communication process in spinal muscular atrophy: A cross-cutting view of the new challenges facing the therapeutic era.

The emerging spectrum of neurodevelopmental comorbidities in early-onset SMA_Baranello_Eur J Paediatric Neurol_2023.pdf

The emerging spectrum of neurodevelopmental comorbidities in early-onset Spinal Muscular Atrophy

Tracker of Policy and Access Environment for SMA Patients-final (1).pdf

Tracker of Policy and Access Environment for SMA Patients

Assessing the policy and access environment accross European counties for SMA patients

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.

SMA Europe Publications and Reports

ASCEND-study-Community-Update-1.pdf

ASCEND study Community Update

In response to your request, we are pleased to share with you information on the ASCEND study, a new global, clinical trial planned to be initiated by Biogen.

Avexis-EC-Approval-Community-Statement_20.05.20-FINAL.pdf

Avexis EC Approval Community Statement

We are delighted to be able to share with you that yesterday, the European Commission (EC)
approved Zolgensma&reg; (onasemnogene abeparvovec), a transformative gene therapy designed to address the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene.

SMA Europe - Community Update Ukraine.pdf

Biogen community statement on Ukraine support

We want to provide an update on Biogen&rsquo;s work to support Ukrainian refugees, including those living with SMA.

21072021_Branaplam-in-SMA_Community-Update-July-2021-FINAL-PDF.pdf

Branaplam in SMA_Community Update

Status of study of branaplam/LMI070 in SMA

Call to Action on JCA.pdf

Call to action on the implementation of the EU Joint Clinical Assessment for ATMPS

FIREFISH Five-Year Update_Cure SMA Research & Clinical Care Meeting 2024.docx.pdf

Community Letter: Roche, FIREFISH 5 year

Community Statement_BIOCAD.pdf

Community Statement BIOCAD July 2023

SMA Community Statement Cure SMA 2023 FINAL.pdf

Community Update from Biogen after Cure SMA 2023 Conference

Rainbowfish - 01_04102023_MR_RB_WMS_En.pdf

Community update from Roche on the RAINBOWFISH clinical trial, for which results from the primary analysis 

Novartis-Acquisition_Community-Statement_15May2018.pdf

Community update – Novartis acquires AveXis

Following the announcement last month, we wanted to let you know that AveXis (the gene therapy company developing a new approach to treat SMA known as AVXS‐101) is officially a Novartis company.

Biohaven Completes Enrollment in Pivotal Phase 3 Study of Taldefgrobep Alfa in Spinal Muscular Atrophy.pdf

Community update: Biohaven Completes Enrollment in Pivotal Phase 3 Study of Taldefgrobep Alfa in Spinal Muscular Atrophy

DEVOTE_study_com_statement-003.pdf

DEVOTE study community statement

The study is designed to evaluate the safety, tolerability, and potentially improved efficacy of nusinersen when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy (SMA).

EMEA-Community-Statement-FINAL-Approved-18MAR2021-pdf.pdf

EMEA Community Statement

As a result of reports from postmarketing safety surveillance that have identified thrombotic microangiopathy (TMA) as a safety signal for onasemnogene abeparvovec, we have updated the Summary of Product Characteristics SmPC to add TMA as a Warning &amp; Precaution.

Community updates

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