News

January 22, 2019

Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA

Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA

Cytokinetics, Incorporated (CYTK) today announced that it has received feedback from the U.S. Food and Drug Administration (FDA) that the Six Minute Walk...

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Topics: Clinical Trials, Research, Front Page News

January 7, 2019

Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam

Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam

On January 7th, PTC Therapeutics, Inc. provided a corporate update, which will be detailed as part of the company's presentation at the 37th Annual J.P. Morgan...

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Topics: Clinical Trials, Research, Front Page News

December 26, 2018

December Webinar on SMA Treatments and Clinical Trials Now Available Online

December Webinar on SMA Treatments and Clinical Trials Now Available Online

A recording of the December 13th webinar updating the community on SMA treatments and clinical trials is now available online. A PDF of the webinar presentation is also available for...

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Topics: Clinical Trials, Research, Front Page 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

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