AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. For more information, ...
WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...
The infantile form of Pompe disease (acid maltase deficiency; glycogen storage disease type II; MIM 232300) is a progressive, lethal metabolic myopathy. Pompe disease is characterized by the massive ...
Now 30, Yu has abandoned her dreams and sold her violin. She can't bear to listen to music, a too-painful reminder of all she lost. A year after the newspaper article appeared, Yu was diagnosed with ...
Researchers at Duke Health have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal ...
Amicus Therapeutics (FOLD) is presenting updated results Wednesday from a small clinical trial involving patients with the rare and debilitating Pompe disease. Amicus’ drug continues to improve ...
Researchers have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal muscle, the liver ...
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