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

Originally published on August 28, 2013. The Cure SMA Scientific Advisory Board (SAB) helps organize, select the content, and chair sessions at the Cure SMA Research Group Meeting. Our SAB has written a summary of the scientific findings from the 2013 meeting.   During the 2013 meeting, 110 research presentations were given, including 38 talks during

Originally published on September 10, 2013. Dr. Mayer and Ms. Battista were awarded funding by Cure SMA for one of four new clinical care focused projects. The project is being conducted at the Children’s Hospital of Philadelphia. This award for $50,000 will fund a pilot study to assess the decision making process by parents of

Originally published on September 17, 2013. Dr. Swoboda was awarded funding by Cure SMA for one of four Clinical Care focused projects. The project is being conducted at the University of Utah. This award for $50,000 will fund a study to determine whether children/preadolescents with SMA type II demonstrate impaired glucose tolerance after glucose loading

Originally published on October 1, 2013. Dr. Timothy Lotze was awarded funding by Cure SMA for one of four new Clinical Care focused projects. The project is being conducted at Texas Children’s Hospital at the Baylor College of Medicine. This award for $50,000 will fund a project to enable health care providers to take better