Meaningful Connections Help Me to See the Possibilities
I didn’t know many kids with Duchenne muscular dystrophy growing up. Everyone around me was physically able; I was the only one who couldn’t ... Read more
Updates
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MDA Awards $389K to UCLA Scientist to Develop Potential DMD Treatment Targeting Sarcospan
The Muscular Dystrophy Association (MDA) awarded $389,000 in funding to a researcher from University of California, Los Angeles (UCLA) to support the development of ... Read more -
European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while ... Read more -
Cell Therapy CAP-1002 Improves Muscle, Lung Function in Boys with DMD, Interim Data Show
CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and lung function in a group of boys and ... Read more -
‘Disabled’ vs. ‘Having a Disability’: Debating the Power of Words
In the disability community, which is as diverse and broad as any other, there is a debate over terminology. I have seen this play ... Read more
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Pfizer’s Gene Therapy Holds Promise in Treatment of DMD, Early Data Show
PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy ... -
UberWAV Is the Next Best Thing for Accessible Transportation
I discovered something different about Washington, D.C. when I moved there for the summer: ... -
Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows
Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including ... -
$500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy
For more than 20 years, Robert (Bob) Bennett was a steadfast leader and supporter ... -
The Speak Foundation Speaks Loudly: A Prelude to September in Chicago
I recently attended The Speak Foundation’s 11th Annual Conference. This event is for individuals ... -
Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow ...
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FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E
News
The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and ... Read more