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FDA Puts 2nd Clinical Hold on IGNITE DMD Trial of Gene Therapy SGT-001 Due to Patient Reaction
NewsOwing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another ... Read more -
Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued
NewsThe clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with Duchenne muscular dystrophy (DMD) has ... Read more -
Attack of the Bloodthirsty Mosquitoes
columns, You, Me and MD - a Column by Leah LeilaniFall and winter have always been my favorite seasons. I eagerly await the cooler weather by prematurely washing my ... Read more -
Local November 9th MDA Disease Education Events Span East, West Coast
NewsThe Muscular Dystrophy Association’s (MDA) efforts to support and advocate for a wide range of neuromuscular diseases are felt ... Read more -
Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says
NewsTreatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved ... Read more -
Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests
NewsThe investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root ... Read more -
Goals Can Simplify Our Complicated Lives
columns, Hawk's-Eye View - a Column by Hawken MillerIt can sometimes feel like everything is against us when doctor appointments and physical barriers bombard us. If we ... Read more -
BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds
NewsAn experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne muscular dystrophy (DMD) in animal ... Read more -
PPMD Grants Funding for Research on Cardiac Health in People with Duchenne
NewsParent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at Nationwide Children’s Hospital and the ... Read more -
Prioritizing Goals in Life with MD
columns, Living, Learning, Thriving - a Column by Ralph YanizI have spent my entire career working and volunteering in the nonprofit arena. My focus has moved from mental ... Read more -
Translarna Preserves Lung Function, Muscle Strength in DMD Boys, Real-World Data Show
NewsTreatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne muscular dystrophy (DMD), real-world data ... Read more -
I Won’t Give Up My Positive Attitude for Others
columns, You, Me and MD - a Column by Leah LeilaniSometimes life with a chronic invisible illness can feel backward and upside down. As if anything that I do, ... Read more
