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Gene therapy delivered early can help children with rare neurodegenerative disease retain motor and cognitive functions

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...
Longview News-Journal

Children's Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy

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 ...

HHS Takes a Lifesaving Step Forward for Newborns in Tribal Communities

When it comes to children born with rare diseases, early diagnosis can make the difference between life and death. Since many ...
News Medical

Gene therapy offers hope for children born with metachromatic leukodystrophy

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...
KXAN

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the ...

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 ...