Things to Keep in Mind When Choosing a Wheelchair
There are many skills to learn if you recently have been diagnosed with a chronic illness. These range from being organized for doctor’s appointments ... Read more
Updates
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College Football Coaches Joining Fight Against DMD During Annual Coach To Cure MD Event
On Saturday, coaches with the American Football Coaches Association (AFCA) across the U.S. will display patches to promote the annual Coach To Cure MD initiative during college ... Read more -
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD ... Read more -
Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A
Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies ... Read more -
Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports
Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a ... Read more
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A Girl’s Best Friend: Thoughts on Mobility Assistance Dogs
One of the best days of my life was when my family and I ... -
Obsessive Compulsive Disorders Evident in Duchenne Patients and in Need of Treatment, Study Says
Obsessive compulsive disorder (OCD), a type of “internalizing disorder,” are evident in children with Duchenne ... -
Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports
Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various ... -
STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide
PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision ... -
Listening and Surrendering to My Body Isn’t Always Easy
Being a healthy able-bodied human being must be nice. Not having to worry about ... -
Numotion, Sing Me a Story Partner to Help Children with Neuromuscular Diseases Turn Life Stories to Songs
Nonprofit Sing Me a Story and Numotion are collaborating to help children with various neuromuscular diseases ...
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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