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National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
News, syndicatedThe EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the ... Read more -
UK Renews Puldysa Early Access Program for Duchenne Patients
NewsOral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be ... Read more -
FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy
NewsSarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) ... Read more -
Don’t Feel Bad About Calling Me ‘Inspiring’
columns, Hawk's-Eye View - a Column by Hawken MillerIn the disabled community, many people discuss the negative implications of being called “inspiring.” Some have said it’s objectifying, ... Read more -
Coronavirus Relief Fund Created to Help DMD Patients, Families
NewsThe COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for ... Read more -
CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk
NewsThe Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label ... Read more -
Looking Backward but Living Life Forward With MD
columns, Living, Learning, Thriving - a Column by Ralph Yaniz“Life can only be understood backwards; but it must be lived forwards,” said philosopher Søren Kierkegaard. He came up ... Read more -
Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer
NewsA Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has ... Read more -
Dating in the Era of Coronavirus
columns, You, Me and MD - a Column by Leah LeilaniIt is undeniable that the coronavirus pandemic has affected many aspects of life. Along with establishing new norms for ... Read more -
Collaboration Provides Cough Assist Machines to British Columbia Residents With Neuromuscular Disease
NewsA partnership between Muscular Dystrophy Canada (MDC) and Technology for Living (TFL) will seek to provide a cough assist ... Read more -
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
cystic fibrosis, News, syndicatedThe Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during ... Read more -
Creativity Allows Me to Surpass Any Physical Limits
columns, Hawk's-Eye View - a Column by Hawken MillerFor a long time, I didn’t think journalism was a creative endeavor. I viewed the craft from an analytical ... Read more
