SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA

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Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient Focused Drug Development (PFDD) Meeting, individuals and families from throughout our community will have the opportunity to testify directly to the FDA. The PFDD meeting will include testimony on a […]

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Cure SMA and Ionis Announce 2016 Holiday Card Contest

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Back by popular demand, Cure SMA and Ionis Pharmaceuticals are once again teaming up for a holiday card contest! This contest is open to children with spinal muscular atrophy, their siblings, and to children of parents with SMA. Ionis will select three entries to use in their annual holiday card. These winning entries will receive

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AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 at the World Muscle Society Congress

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AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an update on interim data from the ongoing Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1 as of September 15, 2016. The data were presented by Jerry Mendell, M.D., director

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Biogen Presents New Data at World Muscle Society Congress

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New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen and Ionis Pharmaceuticals in the late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain. The presentations included safety results from the interim analysis of the Phase 3 ENDEAR study in infantile-onset SMA

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Cure SMA Announces Newborn Screening Initiative in Partnership with MDA

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Twice this year, Cure SMA has had the opportunity to testify before the federal Advisory Committee on Heritable Disorders on the need for newborn screening for SMA. This committee, part of the Health Resources and Services Administration (HRSA), an agency of the U.S. Department of Health and Human Services, determines which conditions will be added

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Biogen and Ionis Release Community Statement on NDA Filing Completion

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Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the the completion of their NDA filing for nusinersen. Dear members of the SMA community, Today we have achieved a crucial step in the pathway to approval of nusinersen. We have completed the submission of our New Drug Application (NDA) to the Food and

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Biogen Completes Rolling Submission of New Drug Application to FDA

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Biogen and Ionis today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for Priority Review which, if granted, would shorten the review period of

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Roche Launches New Phase 2 Clinical Trials

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Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, Roche first announced plans to begin these Phase 2 trials in response to favorable results from a Phase 1 study in healthy volunteers. RO7034067 is an orally available drug that

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Catch Up on Research News from the 2016 Family Friendly Research Poster Session

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Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in slowing or stopping the progress of the disease. With such great promise in the research landscape, we know that no single group can develop a treatment for SMA alone—it requires

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Roche Provides New Update on Clinical Development of RG7916

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Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical development program: Dear SMA community, Roche, together with our collaboration partners PTC Therapeutics and the SMA Foundation, would like to provide you with an update on our SMN2 splicing modifier clinical development program. Please

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