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 […]

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

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

Originally published on August 9, 2013. PTC Therapeutics, Inc. (NASDAQ:PTCT) announced on August 8 the selection of a development candidate in its spinal muscular atrophy (SMA) collaboration with Roche and the SMA Foundation. “We are excited about this important achievement in our SMA program”, stated Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “This

Originally published on August 15, 2013. Dr. Krainer is a Professor at Cold Spring Harbor Laboratory, a member of the Cure SMA Scientific Advisory Board, and a long-term collaborator of Isis Pharmaceuticals.  The grant award to Dr. Krainer’s lab is the fourth drug discovery project funded by Cure SMA in 2013 with a total investment

Originally published on August 22, 2013. Dr. Hua is a Research Investigator at Cold Spring Harbor Laboratory in the laboratory of Dr. Adrian Krainer. He is a long-term collaborator of Isis Pharmaceuticals.  The grant award to Dr. Yimin is the fourth drug discovery project funded by Cure SMA in 2013 with a total investment of