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MDA 2025: Benefits seen for 3 OPMD patients given gene therapy

Summary by Muscular Dystrophy News
The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the first three people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakening in the throat and eyes — in a clinical trial. That’s according to interim data shared at the Muscular Dystrophy Association‘s 2025 MDA Clinical & Scientific Conference, held last month in Dallas. Improved swallowing ability among t…
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MDA 2025: Benefits seen for 3 OPMD patients given gene therapy

The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the first three people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakening in the throat and eyes — in a clinical trial. That’s according to interim data shared at the Muscular Dystrophy Association‘s 2025 MDA Clinical & Scientific Conference, held last month in Dallas. Improved swallowing ability among t…

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Muscular Dystrophy News broke the news in on Tuesday, April 1, 2025.
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