Please provide your email address to receive an email when new articles are posted on . Biotech firm SynaptixBio has entered into a licensing agreement with the Children’s Hospital of Philadelphia, ...
Finding showed treatment with Lenmeldy significantly extended severe motor impairment-free survival in children with PSLI MLD compared with untreated natural history children. The Food and Drug ...
HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...
CINCINNATI (WKRC) - There is promising new research for a genetic condition, which impacts thousands of families worldwide. A small company in the U.K. is giving one mother and son reason to hope. The ...
Metachromatic leukodystrophy is a rare genetic disorder that affects between 1 and 9 people out of 100,000 and manifests itself mainly in childhood and adolescence—with 10 to 20% of cases observed in ...
A genetic medicine at the heart of a $387 million Kyowa Kirin acquisition has won FDA approval, a regulatory decision that makes the one-time treatment the first approved therapy for the rare ...
TOKYO and LONDON and BOSTON, March 18, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients ...
Leukodystrophies are deadly neurodegenerative diseases that affect one in 7,000 children and remain incurable. These genetic diseases attack myelin or the "insulating rubber sheath" surrounding ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA approved ...
This archived news story is available only for your personal, non-commercial use. Information in the story may be outdated or superseded by additional information. Reading or replaying the story in ...
PHILADELPHIA, March 11, 2025 /PRNewswire/ -- In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased ...
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