Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...
New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...
5don MSN
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...
The study uncovers the molecular 'rules' driving the arrangement of emerin into nanoclusters and the mechanisms leading to their defective assembly in people with muscular dystrophy. A new ...
The Associated Press on MSN12d
Patient dies following muscular dystrophy gene therapy, Sarepta reportsWASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for ...
WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%.
Duchenne muscular dystrophy causes progressive muscle degeneration and weakness, but physical exercise should still be part of a comprehensive treatment strategy. Duchenne muscular dystrophy (DMD ...
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