Resources

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

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

Scientific Posters by SMA Europe

CT workshop paper - jnd_221538.pdf

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

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.

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)

Treatment & Clinical Trial resources

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

EUPESMA Survey on SMA and Clinical Trials.pdf

EUPESMA Survey on SMA and Clinical Trials

Publications 

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.

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

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

Branaplam in SMA_Community Update

Status of study of branaplam/LMI070 in SMA

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

Community_Letter_March_2024_Congresses_27 March 2024.pdf

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

Rainbowfish - 01_04102023_MR_RB_WMS_En.pdf

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

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.

190515_External-Message-to-SMA-patient-community_SMA-EUROPE.pdf

External Message to SMA patient community

Enrollment to the ongoing branaplam clinical study is now closed.

JEWELFISH-recruitment-complete-Community-Letter_FINAL.pdf

JEWELFISH recruitment complete Community Letter

As part of our ongoing partnership and following your request to be kept updated on the risdiplam clinical development program, we are pleased to let you know that recruitment for the clinical trial JEWELFISH is now complete.

25112019_MR_FDA_risdiplam_en.pdf

Media Release FDA risdiplam

FDA grants priority review to Roche&rsquo;s risdiplam for spinal muscular atrophy

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-FIREIFSH_Part2_2Years_AAN.pdf

Roche Community Letter FIREIFSH Part 2

We are pleased to share new two-year data from part 2 of the FIREFISH study, which is assessing Evrysdi&trade; (risdiplam) in the treatment of infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA).

Roche_Community_Letter_RF_Recruitment_220222.pdf

Roche Community Letter RF Recruitment

Today we are pleased to announce that worldwide recruitment has completed for the RAINBOWFISH clinical trial, which investigates the efficacy and safety of risdiplam in infants with pre-symptomatic SMA

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-FIREFISH-Trial-Part-2-data-Revised.pdf

Roche FIREFISH Trial Part 2 data revised

As part of our ongoing partnership and following your request to receive important and timely information about the risdiplam clinical development program, we are pleased to share with you an update on part 2 of the FIREFISH clinical trial.

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-SMA_MANATEE_Community_Letter.pdf

Roche-SMA MANATEE Community Letter.pdf

Update on Roche&rsquo;s SMA global clinical development programme: New global combination study to begin in early 2022

SAPPHIRE-Community-Statement.pdf

SAPPHIRE Community Statement

We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular Atrophy (SMA).

SMA-Community-Update-RESPOND-July-2020_final.pdf

SMA Community Update RESPOND

In response to your request, we are pleased to share with you information regarding the RESPOND trial, a new trial which Biogen is planning to initiate.

SMA-Europe-Community-Update-Sept2019-1.docx

SMA Europe Community Update

We are pleased to share with you information regarding the recently presented results of the NURTURE study.

SMA-Europe-Community-Update-DEVOTE-2019_Sept_14092019-002-1.pdf

SMA Europe Community Update DEVOTE

In response to your request for information regarding Biogen&rsquo;s ongoing clinical development programs in spinal muscular atrophy (SMA), we are pleased to share with you information regarding the new global clinical trial DEVOTE.

Resources

Scientific Posters by SMA Europe (1)

Treatment & Clinical Trial resources (15)

Living with SMA: guides & resources (3)

Publications (2)

Community Letters & Updates (30)

Categories

Scientific Posters by SMA Europe (1)

Treatment & Clinical Trial resources (15)SizeFormatUpdated

Treatment & Clinical Trial resources (15)

Living with SMA: guides & resources (3)SizeFormatUpdated

Living with SMA: guides & resources (3)

Publications (2)SizeFormatUpdated

Publications (2)

Community Letters & Updates (30)SizeFormatUpdated

Community Letters & Updates (30)

Categories