News

February 2, 2018

Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday

On Thursday, February 8, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, will vote on whether to recommend newborn screening for spinal muscular atrophy. This decision is an important step toward our goal of having every baby born in the United States...

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Topics: Our Impact, Advocacy, Front Page News

February 1, 2018

AveXis Releases Community Statement on STR1VE

AveXis Releases Community Statement on STR1VE

AveXis has provided the following community statement on AVXS-101.

Dear SMA Community,

AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, is pleased to initiate screening for the remaining patients to be enrolled in the pivotal trial of...

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Topics: Clinical Trials, Research, Front Page News

January 30, 2018

Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy

PTC Therapeutics, Inc. recently announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part seamless, open-label, multi-center study to investigate the safety and efficacy of RG7916 in infants and babies with Type 1 SMA. RG7916 has been safe and well tolerated at all...

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Topics: Clinical Trials, Research, Front Page News

January 26, 2018

AveXis Releases Community Statement on Expanded Clinical Trials

AveXis Releases Community Statement on Expanded Clinical Trials

AveXis has provided the following community statement on AVXS-101. 

Dear SMA Community, 

At AveXis, the gene replacement therapy company developing a new approach to treat SMA known as AVXS-101, we recently announced our plans to start three new clinical...

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Topics: Clinical Trials, Research, Front Page News

January 25, 2018

Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15

Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15

On Thursday, February 15, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST), Cure SMA will hold a webinar updating the community on treatment access and clinical trials. Among the topics covered will be: 

  • The current status of Spinraza access, including dosing, sites, and...

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Topics: Community & Awareness, Clinical Trials, Front Page News

January 19, 2018

SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report

SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report

Cure SMA and our partners in the SMA Industry Collaboration are pleased to announce the release of the Spinal Muscular Atrophy Voice of the Patient (VoP) Report. This report is a thorough written account...

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Topics: Our Impact, Advocacy, Front Page News

January 17, 2018

2018 Updated SMA Drug Pipeline Released

2018 Updated SMA Drug Pipeline Released

We’ve recently released an update to the SMA drug pipeline. This latest version includes:

  • 16 active programs, including one approved therapy. 
  • 14 pharmaceutical partners.
  • 6 programs in clinical trials.
  • An ever-increasing breadth of...

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Topics: Clinical Trials, Research, Front Page News

January 16, 2018

AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for...

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Topics: Clinical Trials, Research

January 9, 2018

Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials

Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials

Scholar Rock, a biotechnology company based in Boston, has announced that they will be investing nearly $50 million to bring SRK-105, a muscle drug for possible treatment of SMA, into human clinical trials. SRK-105 is Scholar Rock’s

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Topics: Clinical Trials, Research, Front Page News

January 5, 2018

Cure SMA Launches Advocacy Action Network

Cure SMA Launches Advocacy Action Network

Working together, we’ve achieved tremendous success in advocating for ourselves, our families, and our whole community, particularly in the past few years.

Today, we’re building on this momentum with the launch of our

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Topics: Our Impact, Advocacy, Front Page News

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