Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar

Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar

<p dir="ltr">Duchenne Muscular Dystrophy is a rare, X-linked neuromuscular disorder that leads to progressive muscle degeneration, loss of ambulation, and premature mortality due to respiratory and cardiac failure. Historically, Duchennke Muscular Dystrophy has been managed through s...

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Bibliographic Details
Main Author: Mahmoud Fawzi Osman (23770893) (author)
Other Authors: Khalid Ibrahim (3853360) (author), Claire Gleeson (23770896) (author), Haytham Ibrahim (21406778) (author), Ikram Ul Haque (23770899) (author), Noora Alhamad (6187880) (author), Tawfeg Ben-Omran (6663634) (author)
Published: 2025
Subjects:
Biological sciences
Genetics
Biomedical and clinical sciences
Neurosciences
Paediatrics
Pharmacology and pharmaceutical sciences
Duchenne Muscular Dystrophy (DMD)
Neuromuscular disorder
Gene therapy
Pediatric neurology
Rare diseases
Clinical outcomes
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