Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...
For more than two decades, researchers at the University of Basel, Switzerland, have been investigating a severe form of muscular dystrophy in which muscles progressively degenerate. The research team ...
A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...
Maine filmmaker living with muscular dystrophy to premiere nature film, proceeds benefiting research
ELLSWORTH, Maine (WABI) - On Saturday, November 22, those who head to The Grand Theater in Ellsworth will get a chance to see the state of Maine in a way they’ve likely never seen before. “Seasons of ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
The family of a boy with a severe degenerative muscle disease has urged their local hospital to prescribe a drug that could help slow down the progress of the condition. Six-year-old Jack was ...
The U.S. Food and Drug Administration (FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three ...
Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...
Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...
Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...
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