News

October 18, 2018

AveXis Issues Community Statement on FDA Filing for SMA Type I

AveXis has provided the following community statement on their FDA filing for SMA type I.

Dear SMA Community,

AveXis, a Novartis company, is pleased to let you know that we have submitted regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important and exciting initial...

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Topics: Clinical Trials, Front Page News

October 18, 2018

AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy
Type I

AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy <br> Type I

AveXis, Inc., a Novartis company, today announced that they have filed for FDA approval of AVXS-101, a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which is missing or mutated in individuals with SMA.

This first filing is for intravenous (IV) delivery of gene...

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Topics: Clinical Trials, Front Page News

October 8, 2018

New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA

New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA

Biogen, Inc. announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25...

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Topics: Clinical Trials, Research, Front Page News

October 5, 2018

Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA

Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA

Cytokinetics, Incorporated and Astellas Pharma Inc. today provided an update for their collaborative skeletal muscle program, including clinical trial updates for reldesemtiv in SMA.

In June, Cytokinetics announced data from a Phase...

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Topics: Clinical Trials, Research, Front Page News

October 3, 2018

Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress

Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and...

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Topics: Clinical Trials, Research, Front Page News

October 3, 2018

Genentech Releases Community Statement on Risdiplam

Genentech Releases Community Statement on Risdiplam

The following statement is a community update from Genentech/Roche on the risdiplam studies FIREFISH and SUNFISH. 

Dear SMA community,

We are happy to share an update on our FIREFISH and SUNFISH studies of risdiplam. We would also like to acknowledge the tremendous support...

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Topics: Clinical Trials, Research, Front Page News

July 10, 2018

Roche Releases Community Update on Risdiplam

Roche Releases Community Update on Risdiplam

The following is a community update from Genentech/Roche on the risdiplam (RG7916) program.

Dear SMA advocacy community,

As requested, we are very pleased to share an update on the RG7916 program.

We would also like to extend a special thanks to study participants and...

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Topics: Clinical Trials, Research, Front Page News

June 19, 2018

PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings

On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy...

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Topics: Clinical Trials, Research, Front Page News

June 16, 2018

Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by...

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Topics: Clinical Trials, Research, Front Page News

June 1, 2018

Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing...

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Topics: Clinical Trials, Research, Front Page News

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