We’ve recently released an update to our SMA drug pipeline. This latest version shows the continued growth of the pipeline, setting several new marks for both breadth and depth: We have 18 active programs. We have 15 pharmaceutical partners. We have seven programs in clinical trials. The cumulative pipeline total is 26 programs, including eight […]
SMA Drug Pipeline Grows to 18 Programs Read More »
The Spring 2015 issue of Directions, our biannual family support newsletter, builds our community by sharing stories and experiences. Families from all over the world connect with each other through Cure SMA to chat, organize, and share stories, photos, news, information, and—most importantly—hope. This issue includes information on the upcoming 2015 Annual SMA Conference, news
Spring 2015 Directions Posted Online Read More »
Biogen, in partnership with Isis Pharmaceuticals, has announced a new Phase 2 clinical trial, called EMBRACE. EMBRACE is a multi-center, Phase 2 clinical study evaluating the safety and exploratory efficacy of the investigational drug, ISIS-SMNRx, (ISIS 396443) in patients with infantile or childhood-onset Spinal Muscular Atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled
Biogen Announces EMBRACE, a New Phase 2 Clinical Trial Read More »
The 2015 Annual SMA Conference Booklet has been posted. The booklet includes: A complete schedule, including information about the special events we’ve got planned for each evening. Times, locations, details, and speaker bios for all of our exciting workshops. Information on our sponsors and exhibitors, who have generously supported this conference. And more! Even if
2015 Annual SMA Conference Booklet Now Posted Read More »
The 15th Annual Concert for a Cure was held on Saturday, May 9, 2015, a the Diablo Country Club in Danville, CA and was a tremendous success raising over $150,000 for a treatment and cure. The evening was made rewarding for donor and families when Dr. John Day, Stanford Professor for Neurology, Pediatrics, and Pathology,
High School Students Lead the Way at Concert for a Cure Read More »
Novartis has published a paper in the journal Nature Chemical Biology, detailing promising results in their program to develop an orally available SMA drug that corrects SMN2 splicing. In testing, the drug increased SMN protein levels and extended survival in a severe mouse model of SMA. Novartis is currently testing this drug in a Phase
Novartis Launches Phase 2 Clinical Trial Read More »
Cure SMA has announced a new partnership with the District Policy Group, a boutique lobbying, public policy, and advocacy practice, to advocate for several key policy areas on behalf of the SMA community. With seven drug programs now in clinical trials, the Patient-Focused Drug Development emphasis of PDUFA V, and the progress toward passage of
Cure SMA Announces Partnership with The District Policy Group Read More »
