The spinal muscular atrophy community is celebrating significant progress toward adding SMA to newborn screening panels in two states. Missouri to Become the First State to Screen for SMA In Missouri, Senate Bill 50 has been passed by both the Missouri House and Missouri Senate, and is currently awaiting the governor’s signature. This bill would […]
SMA Newborn Screening Moves Forward in Missouri and Florida Read More »
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 the Type B Chemistry Manufacturing and Controls (CMC) meeting. This alignment includes support for the proposed commercial manufacturing process, the proposed analytical methods and corresponding
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 health and well-being of individuals and families affected by SMA. The federal Fiscal Year runs from October 1, 2017 through September 30, 2018. Of serious
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 SMA-associated signs and symptoms before 21 months of age; the previous policy’s criteria for treatment of onset symptoms was 6 months or younger. In 2016,
Anthem Updates Insurance Policy for Spinraza Read More »
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 the European Union (EU) for SMA. Spinraza was reviewed under the European Medicines Agency’s (EMA) accelerated assessment program, intended to expedite access to patients with
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 activity of SMN in human cells.” This projects aims to understand the details of how SMN protein works in the cell. Drs. Gruss and Fischer
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 muscle activator, for the potential treatment of spinal muscular atrophy. Orphan designation is granted to drugs and biologic products that are intended for the safe
Cytokinetics Receives FDA Orphan Drug Designation for CK-2127107 for SMA Read More »
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 invested to treating all ages, stages and types of SMA. With such great promise in the research landscape, we know that collaboration between academics, industry, government and families is crucial.
Cure SMA Announces 2017 Family Friendly Poster Session Presenters Read More »
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, for his project, “Epigenetics in SMN2 E7 Alternative Splicing.” Epigenetics refers to changes that affect how much protein is produced from each gene, without altering the DNA sequence. Dr. Kornblihtt
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 the Recommended Uniform Screening Panel (RUSP). The RUSP is a list of conditions that all newborns in the US are recommended to be screened for. The RUSP application for SMA
RUSP Nomination for SMA Accepted into Evidence Review Read More »
