An expression of the live unlimited spirit, stories and voices of the MDA community
MDA Lock-Up Jailbirds can earn their chance to win a pair of roundtrip airline tickets, Beats wireless headphones or an iPad Air 2. For every $100 Jailbirds raise between now and Friday, November 18 they will be automatically entered into the drawing.* Jailbirds are encouraged to track their progress at their Participant Center, and check . . .
This September as we recognize Newborn Screening Awareness month, we are proud to announce a new partnership with Cure SMA aimed at moving newborn screening forward for spinal muscular atrophy (SMA)—the leading genetic cause of death in infants. This is an exciting time for the neuromuscular disease community as decades of research are translating into . . .
Michael Huang, the NHMRC Peter Doherty Postdoctoral Fellow in the department of pathology, Bosch Institute, at the University of Sydney, Australia, was awarded an MDA development grant totaling $177,100 over a period of three years to explore how deficiency of the frataxin protein in Friedreich’s ataxia (FA) may alter the function of cellular power supplies . . .
The investigational drug amifampridine phosphate (brand name Firdapse), under development by Catalyst Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Orphan Drug designation for the treatment of myasthenia gravis (MG). Amifampridine phosphate is a potassium channel inhibitor designed to prolong signals released from nerves and allow greater stimulation of muscles. The drug currently is . . .
Researchers at the National Institutes of Health (NIH) seek children with neuromuscular disorders (NMDs) and/or their caregivers (parent or legal guardian) to complete questionnaires. Researchers would like to develop a tool to better assess the quality of life of people with NMDs, such as muscular dystrophy. The goal of this study is to validate two different . . .
Today we couldn’t be happier as, together with the Duchenne community, we celebrate FDA accelerated approval of eteplirsen to treat kids and adults with some forms of Duchenne muscular dystrophy (DMD). “This is something that we’ve watched, and hoped and prayed for, and seen it develop over the last seven years or so,” said Josh . . .
Bernadette Scarduzio doesn’t like secrets. Her life is an open book — in fact, now it’s a documentary. But Bernadette and her struggles didn’t start out in the spotlight. Her disease was a secret. Born with Charcot-Marie-Tooth Disease or CMT, an inherited disorder which impacts the nerves in the legs and arms, Bernadette was in . . .
Since the tender ago of five, Hugo Trevino, who has Spinal Muscular Atrophy (SMA), has been involved with MDA. From participating in MDA Summer Camp as a child to rallying behind causes like Fill the Boot and Muscle Walk, Hugo has always been dedicated to helping others live longer and grow stronger. Now, as a . . .
September is Charcot-Marie-Tooth Awareness Month. And this month — this year — I’ve never been more aware of CMT. I was diagnosed when I was 19, after a series of falls and half a semester of college note-taking during which I suddenly couldn’t hold a pen. I couldn’t tie my shoes. I struggled with buttons. . . .
Meet Cadence Hometown: Havre, Montana Age: 9 Diagnosis: I have an unspecified myopathy. Myopathies are diseases that cause problems with the tone and contraction of skeletal muscles. Favorite School Subject/Activities: Art, Reading, Rocket Math and Science are my favorite things about school. Favorite People and/or Pets: I love dolphins, including the Miami Dolphins. Although my #1 animal is . . .
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