Latest News
Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy
CK-2127107 is a novel fast skeletal muscle troponin activator being developed as a potential treatment for people living with SMA and certain other debilitating neuromuscular and non-neuromuscular conditions associated with […]
Read More ›Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings
Today, Biogen and Ionis Pharmaceuticals provided a statement regarding the decision to submit the first ever SMA regulatory filings for FDA approval. Dear Members of the SMA Community, Today marks […]
Read More ›Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
Biogen and Ionis have been testing nusinersen in clinical trials and today announced that they will close ENDEAR, the Phase 3 trial testing nusinersen in infants with SMA type 1. […]
Read More ›Cure SMA Announces $2.5 Million in New Planned Research Funding
At the 2016 Annual SMA Conference, Cure SMA announced $2.5 million in new planned research funding over the next 12 months. This funding will be used strategically to help accelerate […]
Read More ›AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted […]
Read More ›Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” in the journal Scientific Reports. […]
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