News

June 6, 2019

Biogen Issues Q2 Community Statement on Spinraza

Biogen Issues Q2 Community Statement on Spinraza

Dear members of the SMA community,

We are grateful for the opportunity to continue to help support individuals with spinal muscular atrophy (SMA), their caregivers and families. We deeply appreciate our shared goals and are dedicated to helping people with SMA, of all ages, and welcome...

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Topics: Clinical Trials, Research, Front Page News

June 4, 2019

Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers

Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers

Scholar Rock today announced positive final top-line results from the Phase 1 clinical trial of its product candidate, SRK-015, a highly specific...

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Topics: Clinical Trials, Research, Front Page News

May 24, 2019

AveXis Issues Community Statement on Zolgensma Approval

AveXis Issues Community Statement on Zolgensma Approval

Dear SMA Community,

We have exciting news to share with you — a major milestone has been reached in the fight against spinal muscular atrophy (SMA)! The US Food and Drug Administration (FDA) has approved ZOLGENSMA® (onasemnogene abeparvovec-xioi) for the treatment of children...

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Topics: Clinical Trials, Research, Front Page News

May 24, 2019

AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age

AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age

AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor neuron 1 (SMN1) gene,...

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Topics: Clinical Trials, Research, Front Page News

May 16, 2019

Novartis Issues Community Statement on Branaplam Clinical Study

Novartis Issues Community Statement on Branaplam Clinical Study

Dear SMA Community,

We are writing to inform you that enrollment to the ongoing branaplam clinical study is now closed.

Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. We are pleased to announce...

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Topics: Clinical Trials, Research, Front Page News

May 8, 2019

Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015

Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015

The below statement is a community update from Scholar Rock on SRK-015. 

Dear SMA Community,

Scholar Rock is dedicated to developing novel medicines to improve the lives of individuals who have Spinal Muscular Atrophy (SMA). We want to thank the SMA community for your...

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Topics: Clinical Trials, Research, Front Page News

May 7, 2019

Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting

Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting

Genentech, a member of the Roche Group, recently announced new data at the 71st American Academy of Neurology (AAN) Annual Meeting from the dose-finding Part 1 of the pivotal...

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Topics: Clinical Trials, Research, Front Page News

May 6, 2019

AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients

AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients

AveXis, a Novartis company, recently announced interim data from ongoing trials of the investigational product...

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Topics: Clinical Trials, Research, Front Page News

May 6, 2019

Biogen Presents Data Affirming Longer-Term Safety and Durability of Treatment with SPINRAZA® at AAN Annual Meeting

Biogen Presents Data Affirming Longer-Term Safety and Durability of Treatment with SPINRAZA® at AAN Annual Meeting

Biogen today announced new data affirming the safety and durability of SPINRAZA (nusinersen) and highlighting its clinically meaningful benefits for...

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Topics: Clinical Trials, Research, Front Page News

May 1, 2019

Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA

Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA

Biogen today announced it will present new data focusing on SMA at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, PA.,...

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Topics: Clinical Trials, Research, Front Page News

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