Author name: Morgan Tuttle

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

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