Moving into the Future: Assistive Technology Can Make a Difference
Many people love to travel. The issue of wheelchair accessibility on airplanes is important. Just as important for those of us who don’t use ... Read more
Updates
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PPMD Awards $100K for Immune Cell Profiling Research for Duchenne and Becker at UCLA
The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the signature profile of immune cells ... Read more -
Choosing the Right Dog Breed for My MD Lifestyle
Prior to my diagnosis, my family spent much more time traveling than we do now. Even though I’ve never traveled outside California, I still ... Read more -
Traveling the World Is Possible, Even with Duchenne
In a past column, I wrote about focusing on the things I can do rather than those I can’t. One of the things I ... Read more -
Sarepta Acquires Rights to Novel Gene Therapy Candidate to Treat Limb-Girdle MD
Sarepta Therapeutics is acquiring an investigational gene therapy program focused on calpain-3 for the treatment of limb-girdle muscular dystrophy type 2A (LGMD2A). The ... Read more
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Let’s Prepare Ourselves for the Trials of a Lifetime
Research and clinical trials Science is closer than ever before to finding treatments and ... -
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — ... -
Duchenne Teen Hits the Runway at Las Vegas Fashion Show
Joey Chorzewski has Duchenne muscular dystrophy, a neuromuscular disease that makes him different from ... -
My Search for a Comfortable Bra as a Woman in a Wheelchair
As women, we reach a point growing up when we must enter the intimates ... -
Video Games Let Me Be Competitive Without the Need to Be Physical
I’m not going to be an athlete — and that’s OK. I’ve found a ... -
#AANAM – FSHD Patients ‘Very Enthusiastic’ About Taking Part in Clinical Trials, Research Suggests
People with facioscapulohumeral muscular dystrophy (FSHD) are “very enthusiastic” about participating in clinical trials ...
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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 Nationwide Children’s ... Read more