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 »
The 15th Annual New England Chapter Walk-n-Roll was held on Saturday, May 16, at beautiful DCR Wompatuck Park in Hingham, MA. The sun was shining as more than 600 people participated in the 1.5 mile walk. Last year, this event hit a milestone by reaching $1 million raised in its history, and this year has
15th Annual New England Walk-n-Roll Raises Over $100,000 Read More »
For the seventh consecutive year, Cure SMA has received a grant from the National Institutes of Health (NIH) for the SMA Researcher Meeting. This year’s grant will go toward conference expenses for fellows, who are younger researchers in training. With the help of this NIH grant, Cure SMA generously subsidizes the cost of registration and
Cure SMA Receives NIH Grant Read More »
Cure SMA mourns the loss of Ken Emerson, volunteer at the Cure SMA National Office. Since his grandson Ryan was diagnosed with SMA type III, Ken became everyone’s favorite visitor at the National Office. Ken even “earned” his own chair at the office, which he was proud to claim as his own! He always put
In Memory of Ken Emerson Read More »
Yesterday, the 21st Century Cures bill was unanimously passed by the House Energy and Commerce Committee. 21st Century Cures would overhaul how the Food and Drug Administration evaluates new medical products and provide $10 billion in mandatory funding for the National Institutes of Health. Passage by the Committee is a significant milestone for this important
21st Century Cures Unanimously Passes Committee Read More »
