Pediatric Rehabilitation Lecture Series: Current and Emerging Genetic Therapies in Dystrophinopathies: DMD-Pediatric Rehabilitation Lecture Series: Current and Emerging Genetic Therapies in Dystrophinopathies: DMD

Pediatric Rehabilitation Lecture Series: Current and Emerging Genetic Therapies in Dystrophinopathies: DMD

Video Transcription

Video Summary

In this video transcript, Dr. Stratton discusses advancements in treating Duchenne muscular dystrophy, focusing on genetic-based therapies such as exon skipping, gene transfer therapy, and CRISPR-Cas9 gene editing. She emphasizes the shift from a palliative to an active treatment approach and the potential for these therapies to improve muscle stability, contractility, and longevity. Dr. Stratton also touches on the importance of ongoing monitoring, potential risks and benefits of the treatments, and the need for comprehensive care for patients with neuromuscular conditions. Additionally, she discusses the role of pediatric rehabilitation in addressing musculoskeletal issues, recommending standing, weight bearing, and joint range of motion exercises to support optimal care for patients with Duchenne muscular dystrophy. Lastly, she highlights the need for continued education and collaboration within the healthcare community to provide effective and individualized care for patients.

Keywords

Duchenne muscular dystrophy

genetic-based therapies

exon skipping

gene transfer therapy

CRISPR-Cas9 gene editing

muscle stability

contractility

longevity

pediatric rehabilitation