News

December 13, 2018

Biogen Issues Q4 Community Statement on Spinraza

Biogen Issues Q4 Community Statement on Spinraza

Bigoen has provided the below community statement on Spinraza. 

Dear Members of the SMA community,

As we approach the two-year anniversary of the U.S. Food and Drug Administration’s (FDA) approval of SPINRAZA® (nusinersen), we want to thank you for your ongoing...

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Topics: Clinical Trials, Research, Front Page News

December 12, 2018

AveXis Issues Community Statement on BLA Acceptance

AveXis Issues Community Statement on BLA Acceptance

AveXis has provided the following community statement on the BLA submission and acceptance for their investigational therapy, AVXS-101 - now known as ZOLGENSMA®.

Dear SMA Community,

We are providing this update to the SMA community in the U.S. at the request of Cure SMA....

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Topics: Clinical Trials, Research, Front Page News

December 3, 2018

Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101

Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101

Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA®...

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Topics: Clinical Trials, Research, Front Page News

November 29, 2018

Register for Cure SMA's Webinar on SMA Treatments and Clinical Trials

Register for Cure SMA's Webinar on SMA Treatments and Clinical Trials

On Wednesday, December 12th, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST) Cure SMA will hold a webinar updating the community on the latest information on SMA treatments and trials. Topics covered will include:

  • AveXis's BLA filing with the FDA
  • Status of access...

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Topics: Support & Care, Clinical Trials, Front Page News

November 29, 2018

Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA

Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA

Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific...

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Topics: Clinical Trials, Research, Front Page 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

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