An expression of the live unlimited spirit, stories and voices of the MDA community
Michelle Murphy is an avid runner. She routinely pounds the pavement through the neighborhoods of Albany, NY. When she needs a change of pace, she runs on the path through the park that skirts the Hudson River. Michelle even hoofs it to and from the gym. You’d never guess that the 24-year-old has Myotonic muscular . . .
BioMarin Pharmaceutical announced May 31 that it is discontinuing development of its exon skipping drug drisapersen. The company recently withdrew its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting that indicated the CHMP intended to issue a negative . . .
Kim Staats, a postdoctoral researcher at the University of Southern California in Los Angeles, was awarded an MDA development grant totaling $180,000 over a period of three years to study potential causes for sporadic amyotrophic lateral sclerosis (ALS). Using a cutting-edge approach to identify genetic contributors in sporadic ALS, Staats has found a new gene . . .
Editor’s note: Ray Spooner, a certified nurse midwife, who has ushered thousands of babies into the world, was diagnosed with ALS just a few months after dousing himself in the Ice Bucket Challenge of Summer 2014. An avid cyclist and fitness enthusiast, Ray knew he had little time before his physical capabilities would be taken from him. Shortly . . .
To say that Harley-Davidson and MDA were made for each other doesn’t fully do the relationship justice. But for two organizations that may seem on their surfaces to have little in common, there are a number of uncanny similarities. One of them I continue to be surprised by is the spirit of living unlimited. Harley . . .
The investigational drug omigapil, under development by Santhera Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of congenital muscular dystrophy (CMD). Preclinical studies in disease models have shown the drug inhibits cell death and reduces body weight loss and skeletal deformation while increasing mobility and improving . . .
The Informed Consent Authorization Form is 12 pages long. My husband, Allen Carney, reads it carefully, initials in several places and signs at the end. And that’s the easiest part of participating in a research project. Welcome to the world of giving your time and body to research for Facioscapulohumeral muscular dystrophy. My husband Allen, . . .
Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) is continuing review and internal discussions related to its New Drug Application for eteplirsen and will not complete the work by the goal date of May 26. Eteplirsen is an exon skipping drug designed to slow the progression of Duchenne muscular dystrophy (DMD) . . .
Hailey Hometown: Bismarck, ND Age: 16 Diagnosis: In 2001, I was diagnosed with Spinal Muscular Atrophy (SMA) Type 2. I was just 2 years old. SMA is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Favorite School Subject/Activities: When I am in school, I like English & Biology class the . . .
Kathrin Meyer, a postdoctoral researcher at Nationwide Children’s Hospital in Columbus, Ohio, was awarded an MDA development grant totaling $180,000 over three years to study the roles of cell types other than motor neurons in amyotrophic lateral sclerosis (ALS). Please describe your current research. For a long time, ALS research focused mainly on the motor . . .
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