Front Page 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 […]
Read More ›Cure SMA Advocacy Activities for the 2017 Year-to-Date
The first half of 2017 has been an active six months for Cure SMA and SMA advocates. Cure SMA and members of our community have […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›Cure SMA Announces 2017 Family Friendly Poster Session Presenters
We’ve recently seen significant advances in SMA research come to fruition, and with 17 drug programs in development, including five in clinical trials, we are […]
Read More ›Cure SMA Awards $140,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires, Argentina
Cure SMA and FAME (Families of SMA, Argentina) have awarded a $140,000 research grant to Alberto Kornblihtt, PhD, at the Universidad de Buenos Aires, Argentina, […]
Read More ›RUSP Nomination for SMA Accepted into Evidence Review
The Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC) announced that they have accepted spinal muscular atrophy into the review process for […]
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