Originally published on September 19, 2013. Isis Pharmaceuticals, Inc. announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA), show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in […]

Originally published on September 4, 2013. Reaching the first milestone was triggered by completion of specific program activities and coincides with the successful completion of all transition obligations to Pfizer by Repligen. Click here for the Repligen press release. Repligen Corporation announced today that it has achieved a milestone from Pfizer, Inc. under the terms

Originally published on January 22, 2014. PTC announces that its SMA drug program, partnered with Roche, begins Phase I Safety Trials in healthy, adult volunteers. This brings the total number of SMA programs in clinical development to 5. PTC Therapeutics, the SMA Foundation, and Roche announced today that their joint research program in SMA has

Originally published on February 20, 2014. Cure SMA is proud to announce our new basic research funding for 2014. We are leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component. Basic research helps us understand what causes SMA, by revealing new and

Originally published on February 27, 2014. Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component.  Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Drs. Laurent and Lutz from Jackson Laboratory

Originally published on March 4, 2014. Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component.  Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Dr. Burnett of the Uniformed Services University

Originally published on March 6, 2014. A critical way Cure SMA advances research is by hosting and financially supporting The SMA Research Group Meeting. The meeting is the largest SMA research conference in the world.  For 18 years, Cure SMA has hosted the conference with the goal of bringing the leading researchers from around the

Originally published on March 11, 2014. Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component. Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Dr. Ebert at the Medical College of

Originally published on March 19, 2014. Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component. Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Dr. Ke-Jun Han at the University of

Originally published on March 25, 2014. Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component.  Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Dr. Rashmi Kothary at The Ottawa Hospital