The US Senate is currently working on legislation to modify the Affordable Care Act. Though details of the Senate’s plan are not yet available, legislation could be released as early as Thursday or Friday of this week. As Senators consider various options it is important that they keep in mind the needs of people such […]
Cure SMA Update – Senate Healthcare Bill Read More »
Cure SMA is pleased to announce the launch of Phase 2 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that
Launch of Phase 2 SMA Industry Collaboration Announced Read More »
The 2017 Annual SMA Conference Booklet is now posted. The booklet includes: A complete schedule with information about the special events we’ve got planned including: the Family Friendly Research Poster Session, Saturday Night at Magic Kingdom Park and Sunday’s Newborn Screening Symposium Information on Saturday afternoon panels focusing on SMA drugs in development and drug
2017 Annual SMA Conference Booklet Now Posted Read More »
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 first half of 2017 has been an active six months for Cure SMA and SMA advocates. Cure SMA and members of our community have been working with stakeholders groups across multiple settings to ensure our patients’ voices are heard. Below is a summary of the key issues and audiences we’ve targeted in our engagement
Cure SMA Advocacy Activities for the 2017 Year-to-Date Read More »
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
