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 »
We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers the second half of a session on SMN Partners and Therapeutic Targets. The session was moderated by Rashmi Kothary, PhD. SMN Partners and Candidate Therapeutic Targets, Part
2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 2 Read More »
Originally published on January 18, 2013. Cure SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies. Dr. Rubin’s project at Harvard University is the first of three drug discovery awards that will be given
Originally published on January 3, 2013. Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Cure SMA. Cure SMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This
Pfizer Licenses Cure SMA Quinazoline Drug Program from Repligen Read More »
