Latest News
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 […]
Read More ›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-015, a muscle drug for possible treatment of SMA, into […]
Read More ›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 […]
Read More ›Minnesota Becomes Second State to Adopt Newborn Screening for SMA
On December 27, the Minnesota Commissioner of Health accepted the recommendation of the state’s Advisory Committee on Heritable and Congenital Disorders to add SMA to Minnesota’s newborn screening panel. With […]
Read More ›Cure SMA Announces Funding for Care Centers Across the US
Cure SMA is pleased to announce the first grants from $450,000 in funding to help increase capacity at SMA treatment sites across the US. In order to increase the number […]
Read More ›Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
Biogen and Ionis Pharmaceuticals announced that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA). Biogen […]
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