Advocacy
Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” […]
Read More ›Cure SMA Posts Keynote Presentations on the NDA Process
The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are […]
Read More ›2016 Update on SMA-FDA Interactions
The SMA drug pipeline has grown dramatically in just over a decade. Of the 18 programs in the pipeline, six of these are in clinical […]
Read More ›Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals
Cure SMA is featured by Astellas Pharmaceuticals as part of their new Changing Tomorrow Together website. Cure SMA Research Director Jill Jarecki contributed a spotlight […]
Read More ›Global Genes Features Cure SMA in Patient Voice Project
Global Genes recently published, “From Molecules to Medicines: How Patients Can Share Their Voices Through the Drug Development Process.” This toolkit was the product of […]
Read More ›Senate HELP Committee Passes Another 7 Innovation Bills
The Senate Health, Education, Labor and Pensions (HELP) Committee yesterday passed an additional 7 bills out of committee. Along with the 7 bills previously passed […]
Read More ›Senate HELP Committee Passes Innovation Bills
The Senate Health, Education, Labor and Pensions (HELP) Committee has passed 7 bills out of committee as part of its “step-by-step” approach to Innovation and […]
Read More ›Update on 21st Century Cures/Innovation: the Senate’s “Step-by-Step” Approach
Cure SMA has been carefully monitoring the consideration of 21st Century Cures on Capitol Hill. As you know, the House passed legislation in July last […]
Read More ›Cure SMA Testifies Before FDA at PDUFA Meeting
The Prescription Drug User Fee Act (PDUFA), the law that allows the FDA to collect fees from drug manufacturers to fund the new drug approval […]
Read More ›5th Annual Congressional Dinner Brings Together Families, Government and Industry
On December 1, more than 120 people from the SMA community—including government, and industry partners—gathered for the 5th Annual “Hope on the Hill” Congressional Dinner. […]
Read More ›