Why Multidisciplinary Care Matters

Clara was 2 years old when she was diagnosed with congenital muscular dystrophy. Her family was referred to their local Minneapolis MDA Care Center, and when they got there, Clara’s mom, Becky, says it felt “a bit like coming home.” Our MDA Care Center is a huge gift for us as working parents managing a . . .

Read More

Simply Stated: What is Muscular Dystrophy?

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 . . .

Read More

MDA Grants Work to Find Breakthroughs Across Diseases

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 . . .

Read More

New Research Grants Aim to Help MDA Families Live Unlimited

  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 . . .

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

Meet Payton, MDA’s 2016 State Goodwill Ambassador from Ohio

Payton Hometown: Cincinnati, Ohio Age: 13 Diagnosis: Congenital Muscular Dystrophy (CMD), a group of muscular dystrophies that become apparent at or near birth. CMD results in overall muscle weakness with possible joint stiffness or looseness. Favorite School Subject/Activities: Reading, writing, and art. Favorite People and/or Pets: I live with my older brother Jacob and my mom, Angela. I also . . .

Read More

Five Questions with CMD Researcher Liza Pon

Liza Pon, professor in the department of pathology and cell biology at Columbia University in New York was awarded an MDA research grant totaling $300,000 over three years to study the underlying mechanisms at work in CHKB congenital muscular dystrophy (CMD). Pon also will test whether therapies that promote function of a protein known as . . .

Read More