Latest News
AveXis Announces Alignment with the FDA on Company’s GMP Commercial Manufacturing Process for AVXS-101
AveXis today announced alignment with the U.S. Food and Drug Administration (FDA) on the company’s Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following the receipt of minutes from […]
Update: President’s Budget Proposes Significant Cuts to Federal Health Programs That Are Critically Important to SMA Patients and Families
The President recently released his detailed budget proposal for Fiscal Year 2018, which proposed significant funding cuts to numerous federal programs, including many that have a direct impact on the […]
Anthem Updates Insurance Policy for Spinraza
Anthem Inc. recently announced an updated insurance policy for the use of Spinraza (nusinersen) for treatment of spinal muscular atrophy. The revised policy expands criteria for treatment to onset of […]
European Commission Grants Marketing Authorization for Spinraza, Making it First Approved SMA Treatment in the European Union
Biogen announced today that the European Commission (EC) has granted a marketing authorization for Spinraza for the treatment of spinal muscular atrophy (SMA), making it the first approved treatment in […]
Cure SMA Awards $140,000 Grant to Oliver Gruss, PhD, Rheinische Friedrich-Wilhelms-Universitat Bonn and Utz Fischer, PhD, Julius-Maximilians-Universitat Wurzburg, Germany
Cure SMA has awarded a $140,000 research grant to Drs. Oliver Gruss and Utz Fischer at Rheinische Friedrich-Wilhelms-Universitat Bonn and Julius-Maximilians-Universitat Wurzburg in Germany to study “Regulatory cues modulating the […]
Cytokinetics Receives FDA Orphan Drug Designation for CK-2127107 for SMA
Cytokinetics, Inc. recently announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK-2127107, a next-generation fast skeletal […]