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Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows
NewsTreatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more ... Read more -
$500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy
NewsFor more than 20 years, Robert (Bob) Bennett was a steadfast leader and supporter of the Muscular Dystrophy Association ... Read more -
The Speak Foundation Speaks Loudly: A Prelude to September in Chicago
columns, Living, Learning, Thriving - a Column by Ralph YanizI recently attended The Speak Foundation’s 11th Annual Conference. This event is for individuals with neuromuscular diseases and their ... Read more -
Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
NewsThe challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were ... Read more -
Potential DMD Treatment, Pamrevlumab, Helps to Preserve Lung, Heart and Upper Limb Function in Youth, Early Trial Data Show
NewsEarly trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or even improve, ... Read more -
Here’s How I Explain My Experience with Duchenne to Others
columns, Hawk's-Eye View - a Column by Hawken MillerDuchenne muscular dystrophy is not easy for people to wrap their heads around. Most haven’t heard of the disease, ... Read more -
MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy
NewsThe Muscular Dystrophy Association (MDA) has awarded Locana $550,000 through its MDA Venture Philanthropy (MVP) program to help advance the biotech ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
NewsIt wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed ... Read more -
Raising Levels of Protein, Sarcospan, Seen to Benefit Cardiac Muscles in Mouse Model of DMD
NewsIncreasing the amount of a protein called sarcospan may help to protect against heart failure in children with Duchenne ... Read more -
You Can Experience the Beauty of the Outdoors, Even with Duchenne
columns, Hawk's-Eye View - a Column by Hawken MillerIt’s hard to find time to spend outside, especially since I have a disease that hampers my mobility, but ... Read more -
Enrollment Progressing Quickly in PolarisDMD Trial Testing Edasalonexent, Catabasis Says
NewsEnrollment is progressing quickly in Catabasis Pharmaceuticals‘ pivotal Phase 3 PolarisDMD trial, testing its investigational small molecule therapy edasalonexent ... Read more -
Edasalonexent, Potential DMD Therapy in Phase 3 Trial, Seen to Preserve Bone Health in Test in Mice Against Corticosteroid
NewsCatabasis Pharmaceuticals announced new preclinical data showing that, in a mouse model of Duchenne muscular dystrophy (DMD), the small molecule ... Read more
