The following is a community update from Genentech/Roche on the risdiplam (RG7916) program. Dear SMA advocacy community, As requested, we are very pleased to share an update on the RG7916 program. We would also like to extend a special thanks to study participants and their families for their incredible commitment to advancing the progress of […]
Roche Releases Community Update on Risdiplam Read More »
Cure SMA has awarded a $300,000 preclinical drug discovery grant to Umrao Monani, PhD, at Columbia University, for his project, ” Restoring function at the NMJ: A novel means to treat SMA.” A drug directed at SMN enhancement, Spinraza, has recently become available but is unlikely to benefit all patients with maximal effectiveness. The objective
Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University Read More »
Health and Human Services Secretary Alex Azar today approved the recommendation that newborn screening for spinal muscular atrophy be implemented nationwide. This recommendation was issued on February 8, 2018, by the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). With Secretary Azar’s signature, SMA becomes part of the Recommended Uniform Screening Panel (RUSP),
HHS Secretary, Alex Azar, Recommends Nationwide Newborn Screening for SMA Read More »
Earlier this month, the New Jersey State Senate Committee on Health, Human Services, and Senior Citizens unanimously approved S. 974. Introduced by Senator Troy Singleton, the bill would add SMA to the New Jersey’s screening panel, and provide follow up information to the family of an affected newborn. Special thanks to SMA community advocates Denise
SMA Newborn Screening Moves Forward in New Jersey Read More »
Thank you to everyone who attended the 2018 Annual SMA Conference at the Hilton Anatole Hotel in Dallas, Texas! It was an impactful weekend of community, research and fun. While the final numbers are still being counted, we are happy to announce that, including on-site registration, there were around 1,600 attendees in total – our
Thank You For a Wonderful 2018 Annual SMA Conference! Read More »
The summer 2018 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced ten grants totaling $1,325,000 million for basic research. This issue of Compass introduces our grantees and gives a brief overview of their projects, including how the research will be conducted and what impact the
Summer 2018 Issue of Compass Now Available Read More »
On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy (SMA), at the 22nd Annual SMA Researcher Meeting. The data presented by Dr. Baranello demonstrated that at Day 182, over 90% of the babies
Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by John W. Day, M.D., Ph.D., Professor of Neurology and Pediatrics (Genetics), Stanford University, at the 2018 Annual Cure SMA Conference in Dallas. This hypothesis-generating study met its primary objective to determine potential pharmacodynamic
Yesterday, Biogen announced it will present data from its SPINRAZA clinical development program –the largest of its kind, with more than six years of data–for spinal muscular atrophy (SMA) at the Cure SMA 2018 Annual SMA Conference in Dallas, TX. “We are proud to support Cure SMA in advancing scientific understanding of the disease to
Biogen to Present New Data at the 2018 Annual SMA Conference Read More »
The 2018 Annual SMA Conference, now in its 30th year, is set to kick off on June 14 and continue into a weekend full of programs and events that bring families, researchers and healthcare professionals together. This year’s conference will be the largest in the Midwest to date. We’re expecting 1,600 attendees, including over 400
The 2018 Annual SMA Conference is Here! Read More »
