Author name: Sarah McCall

FDA Issues Statement on Data Accuracy Issues with Recently Approved Gene Therapy

Leave a Comment / Front Page News /

Today, the FDA issued the below statement identifying data accuracy issues with AveXis’ SMA therapy, Zolgensma. Cure SMA is investigating this issue and will report when we have further details. We will be issuing updates to the community as we have news to share. The FDA’s statement reads: As a public health agency, we believe that it is […]

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Biogen Issues Community Statement on Spinraza

Leave a Comment / Clinical Trials, Front Page News /

Biogen has provided the following community statement on Spinraza. Dear members of the SMA community, Following a busy spring of data presentations at medical congresses and the annual Cure SMA Conference, we are pleased to provide updates on research highlighting SPINRAZA® (nusinersen) data and real-world experience for individuals with spinal muscular atrophy (SMA). Recent updates

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SMA Community’s Voice Heard “Loud and Clear” at Last Week’s Patient Focused Drug Development Meeting with the FDA

Leave a Comment / Community Awareness, Front Page News, Our Impact /

On April 18, 2017, the SMA community—families, clinicians, researchers, industry and regulators—gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA. As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA), the FDA is required to gather community feedback more systematically, through events such as this PFDD meeting, and incorporate that

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