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Mom urges state to add Duchenne muscular dystrophy to newborn screening panel
WTHR Indianapolis· 2 days agoDuchenne muscular dystrophy affects at least 1 in 5,000 male births each year, and girls can be...
Duchenne Muscular Dystrophy: the global clinical trials landscape 2024
Pharmaceutical Technology via Yahoo Finance· 6 days agoAffecting muscle and skeletal strength, this rare genetic disorder impacts thousands of men...
Investigational FSHD Drug Did Little to Change DUX4 Expression
MedPage Today· 3 days agoThe investigational drug losmapimod did not significantly change expression of the gene behind...
TIME100 Health: Jerry Mendell
Time via Yahoo News· 3 days agoJerry Mendell Credit - Courtesy Nationwide Children’s Hospital In the 1960s, Dr. Jerry Mendell saw his first patient with Duchennemuscular dystrophy....
Young boy with Muscular Dystrophy gets surprise from Dothan Police on birthday
WDHN Dothan via Yahoo News· 5 days agoDOTHAN, Ala (WDHN) — A young boy turning six this past weekend got a special surprise from the boys...
Parent Project Muscular Dystrophy Awards $250,000 to Support Clinical Research Network for Duchenne...
FOX 59 Indianapolis· 5 days agoParent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy</ ...
Teenager with rare disease getting set to compete in 13th Flying Pig Marathon
WLWT-TV Cincinnati· 1 day agoFor the 13th year in a row, a teenager who is battling a rare disease is planning to cross the...
Local firefighters to participate in annual Fill the Boot campaign for Muscular Dystrophy...
Arizona Daily Sun· 4 days agoThe International Association of Fire Fighters (IAFF) has collected funds in communities since 1954...
They have jobs but still need help to feed their families
WKOW 27 Madison· 12 hours agoPrior to the Covid-19 pandemic, many were elderly or disabled people on fixed incomes, said Kathleen...
The FDA and Gene Therapy for Duchenne Muscular Dystrophy
Journal of the American Medical Association· 4 days agoThe US Food and Drug Administration (FDA) grants accelerated approval that includes the standard of a surrogate outcome that is “reasonably likely” to predict clinical benefit.1 Gene therapy ...