Front Page News
Cure SMA and Ionis Announce 2016 Holiday Card Contest
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 […]
Read More ›AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 at the World Muscle Society Congress
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 […]
Read More ›Biogen Presents New Data at World Muscle Society Congress
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 […]
Read More ›Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
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 […]
Read More ›Biogen and Ionis Release Community Statement on NDA Filing Completion
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 […]
Read More ›Roche Launches New Phase 2 Clinical Trials
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, […]
Read More ›Catch Up on Research News from the 2016 Family Friendly Research Poster Session
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 […]
Read More ›Roche Provides New Update on Clinical Development of RG7916
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 […]
Read More ›2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. […]
Read More ›Cure SMA Welcomes Aboard New Board and Committee Members
Recently, Cure SMA confirmed three new board members and one new committee member. These new members will provide additional leadership in the areas of advocacy, […]
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