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 . . .
2017 MDA Muscle Walk t-shirt design voting is now live! Now’s your chance to cast your vote for your favorite design for the 2017 MDA Muscle Walk t-shirt! Voting ends midnight on Friday, September 16. Ask your family and friends to vote for their favorite design to help push your selection to the top! The . . .
In 1986, the International Association of Firefighters completed a permanent tribute to its members, the IAFF Fallen Firefighter Memorial, located in Colorado Springs, Colorado. The Memorial was founded to recognize and honor past and future professional firefighters and emergency medical personnel who are killed in the line of duty and to provide assistance to the . . .
