The Muscular Dystrophy Association is pleased to announce the award of a clinical research network grant totaling $918,000 over three years to spur advances in myotonic dystrophy (DM) research.
Ionis Pharmaceuticals provided an update on its recently-concluded phase 1-2 clinical trial of IONIS-DMPK-2.5Rx, an experimental compound for the treatment of type 1 myotonic dystrophy (DM1). Analysis showed mixed results from the trial.
Five Questions with Researcher Matthew Disney Matthew Disney, a professor at the Scripps Research Institute in Jupiter, Fla., was awarded an MDA research grant totaling $300,000 over a period of three years to optimize two novel drug-like compounds — one that targets toxic RNA in myotonic dystrophy and another that targets toxic RNA in ALS . . .
Muscular dystrophy is a term that refers to a number of diseases that cause progressive loss of muscle mass resulting in weakness and, sometimes, loss of mobility. There are many different kinds of muscular dystrophy, each affecting different groups of muscles. In some types of muscular dystrophy, symptoms begin in childhood. In other forms, symptom . . .
Kayla living unlimited by running on the track
Fabiola and Matthew watched their daughter run one lap. Then two. Then three. Kayla rounded the same bend again and again, a look etched on her face that her parents knew well — determination.
All in all, the 10-year-old ran nine laps at the Grand Rapids, Mich., Juniors River Bank Run. Other kids may have run more, but to Kayla and her parents, those nine laps were a marathon. They were as big as beating Usain Bolt in the 100-meter.
Since its inception, MDA has invested more than $1 billion in neuromuscular disease research to uncover new treatments and cures. In 2016 alone, MDA awarded 66 new research, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the . . .
This summer MDA challenged our families, friends, sponsors, staff and others around the nation to share their stories about living life without limits despite the challenges of neuromuscular disease. Some shared moments tied to pursuing an education or career that was thought to be out of reach. Some talked about skydiving, hiking or running a . . .
Steven Markus, assistant professor at Colorado State University in Fort Collins, Colo., was awarded an MDA research grant totaling $300,000 over a period of three years to study alterations in the dynein gene and their effects in spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). His work to elucidate the molecular basis for dynein . . .
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 . . .
Kourtney Rivers, Age 13 Severn, MD Home and Family Kourtney lives with her mom Jackie, dad Tom, and their 2 dogs, Ruby and Lily. Diagnosis Kourtney was diagnosed when she was a year old with Myotonic Muscular Dystrophy, a form of muscular dystrophy that affects muscles and many other organs in the body. MMD causes . . .
