Originally published on September 25, 2013. Dr. Matthew Halanski was awarded funding by Cure SMA for one of four Clinical Care focused projects. The project is being conducted at the University of Wisconsin. This award for $50,000 will fund a project to create a database of patients with SMA from multiple clinical centers.  It will […]

Originally published on December 30, 2013. Cure SMA is proud to have contributed funding to the research published in the following journal articles in 2013. Cure SMA has invested over $55 Million in research to accelerate identification of a treatment and cure for SMA.  This includes 25 research projects in 2013 alone.   Please see the

Originally published on October 24, 2013. Cure SMA invests in four research areas:  Basic Research, Drug Discovery, Clinical Research, and Care Research. Cure SMA has invested over $55 Million in research since 1984, with $35 Million in the past decade alone. In 2013, we provided funding to 25 different research projects across these four areas. We

Originally published on November 5, 2013. Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA). Outcome data are expected to be available towards the

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