Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

26.2 Miles For a Cure: Runner With Muscular Dystrophy Joins Team Momentum Ahead of Chicago Marathon

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 . . .

Read More

Development of Drisapersen and Follow-On Compounds Discontinued

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 . . .

Read More

Five Questions with ALS Researcher Kim Staats

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 . . .

Read More

An Intimate Look at a Life with ALS: Photos of a Marriage, a Family and — of Course — Love

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 . . .

Read More

Santhera’s Congenital Muscular Dystrophies Drug Omigapil Receives FDA Fast Track Designation

    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 . . .

Read More

Participating in Clinical Research Means Giving of Yourself — Literally — to Science

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, . . .

Read More

Five Questions with ALS Researcher Kathrin Meyer

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 . . .

Read More