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 »
Originally published on April 4, 2013. Award to support testing of Tirasemtiv in mouse models of Spinal Muscular Atrophy. Cytokinetics, Incorporated (Nasdaq: CYTK) and Cure SMA announced the award of a grant from Cure SMA to Cytokinetics to support preclinical research on muscle function in a mouse model of spinal muscular atrophy (SMA) to be
Originally published on April 23, 2013. Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced in April the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The study, which will begin enrolling patients soon, is a Phase 2 study in eight patients with infantile-onset SMA. The study, which initiates the Phase
ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy Read More »
Originally published on May 2, 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. This is the third of four drug discovery project awarded by Cure SMA in 2013 with a total
Originally published on May 21, 2013. Results from the Phase II Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANT SMA) clinical trial led by Dr. John Kissel at The Ohio State University have been published in the journal Muscle & Nerve. This study was funded by Cure SMA. The study was a randomized,
Originally published on August 8, 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. This is the fourth drug discovery project funded by Cure SMA in 2013 with a total investment of
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 a session on SMN Partners and Therapeutic Targets. The session was moderated by Arthur Burghes, MD, PhD. SMN Partners and Candidate Therapeutic Targets, Part 1 Individuals with
2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 1 Read More »
