Cure SMA-funded researchers Rebecca Hurst Davis and Kathy Swoboda have published a paper, “Responses to Fasting and Glucose Loading in a Cohort of Well Children with Spinal Muscular Atrophy Type II” in the journal Pediatrics. Elizabeth A. Miller and Ren Zhe Zhang also co-authored the paper. This study was funded by an initial $50,000 Cure
Cure SMA-Funded Researchers Publish Paper in Journal Pediatrics Read More »
Isis Pharmaceuticals, Inc. announced that it has initiated an open-label extension study, SHINE. The SHINE study provides ISIS-SMNRx to infants and children with spinal muscular atrophy (SMA) who have completed their participation in the Phase 3 ENDEAR and CHERISH studies and are eligible to participate in SHINE. All patients in the SHINE study will receive
Isis Pharmaceuticals Announces SHINE, an Open-Label Extension Study Read More »
On Monday, November 9, Cure SMA will be hosting a one-hour webinar on clinical trials and regulatory topics. The webinar will be held at 11:00am CST (9:00am PST/10:00am MST/12:00pm EST). Jill Jarecki, Research Director at Cure SMA, will be joined by a panel of three drug development experts to discuss clinical trial design, expanded access,
Cure SMA to Host Clinical and Regulatory Webinar Read More »
PTC Therapeutics, in partnership with Roche and the SMA Foundation, recently announced new results from their ongoing Phase 2 trial of RG7800, a small molecule splicing modifier for SMA. The presentation was made at last week’s World Muscle Congress in the United Kingdom. Results from the first cohort of patients enrolled in the Phase 2
PTC Announces New Clinical Trial Results at World Muscle Congress Read More »
Biogen and Isis Pharmaceuticals recently provided an update on the clinical development of ISIS-SMNRx, which is currently being tested in Phase 3 clinical trials: Dear members of the SMA community, Over the past few months, we have received many questions from members of the SMA community about ISIS-SMNRx and when it might be widely available
Community Update on ISIS-SMNRx from Biogen and Isis Read More »
Nationwide Children’s Hospital/AveXis recently announced new results from their ongoing Phase 1 trial of systemic gene therapy for SMA. Dr. Jerry R. Mendell, Director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital (NCH), made the presentation at last week’s World Muscle Congress in the United Kingdom. Dr. Mendell, lead
New Gene Therapy Results Announced at World Muscle Congress Read More »
Registration is now open for industry and academic researchers to attend our 2016 SMA Researcher Meeting, which will be held June 16-18 in Anaheim, CA, at the Disneyland Hotel. The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. The 2015 SMA Researcher Meeting set a record, with over
Registration Open for the 2016 SMA Researcher Meeting Read More »
The House of Representatives has passed the Ensuring Access to Clinical Trials Act of 2015 (EACT). This bill already passed the Senate in July, and will now go to President Obama to be signed into law. This bill allows individuals to receive up to $2,000 as compensation for participating in a clinical trial, without those
House of Representatives Passes Ensuring Access to Clinical Trials Act Read More »
A note from Rich Rubenstein, Kenneth Hobby, and the entire Cure SMA team: Cure SMA has had the honor to have Jessica Moyer and her family as one of our volunteer chapter leaders for almost 14 years. Jessica has been critical to the success of our organization, thanks to her efforts locally and nationally. Cure
Thank You to Jessica Moyer and Family Read More »
Thanks to our vibrant community, August was one of our largest and best awareness months ever, with unprecedented levels of public awareness and outreach for SMA. Toward the end of August, we challenged our community to seize this momentum, and convert awareness into action and advocacy. One of these opportunities to convert awareness into action
FDA Engagement Update: The Voices of SMA Read More »
