Digital outcome measures in Duchene muscular dystrophy: Lessons learnt from clinical trials DOI Creative Commons
Camila Gonzalez-Barral, Laurent Servais

Journal of Neuromuscular Diseases, Journal Year: 2024, Volume and Issue: unknown

Published: Dec. 8, 2024

Duchenne muscular dystrophy is a severe neuromuscular disorder characterized by progressive muscle degeneration resulting from mutations in the dystrophin gene. Digital outcome measures offer promising alternative to traditional used clinical trials. This review explores development and application of digital dystrophy, emphasizing feasibility, reliability, sensitivity, validity these measures. The stride velocity 95th centile has been validated as robust endpoint approved for use evaluation drugs treatment European Medicines Agency. Although have potential enhance efficiency accuracy trials, challenges such limited sample sizes patient compliance persist. integration artificial intelligence into data analysis progress, but further validation required before strategies can be incorporated future trial methodologies.

Language: Английский

Hotspots and trends in satellite cell research in muscle regeneration: a bibliometric visualization and analysis from 2010–2023 DOI Creative Commons
Nan Huang, Kang Zou,

Yan-Biao Zhong

et al.

Heliyon, Journal Year: 2024, Volume and Issue: unknown, P. e37529 - e37529

Published: Sept. 1, 2024

Language: Английский

Citations

0
Cell transplantation-mediated dystrophin supplementation efficacy in Duchenne muscular dystrophy mouse motor function improvement demonstrated by enhanced skeletal muscle fatigue tolerance DOI Creative Commons

Clémence Kiho Bourgeois Yoshioka,

Nana Takenaka-Ninagawa,

Megumi Goto

et al.

Stem Cell Research & Therapy, Journal Year: 2024, Volume and Issue: 15(1)

Published: Sept. 19, 2024

Language: Английский

Citations

0
Genomic insights into Duchene muscular dystrophy: Analysis of 1250 patients reveals 30% novel genetic patterns and 6 novel variants DOI Creative Commons
Khalda Amr, Nagia Fahmy, Ghada El‐Kamah

et al.

Journal of Genetic Engineering and Biotechnology, Journal Year: 2024, Volume and Issue: 22(4), P. 100436 - 100436

Published: Nov. 11, 2024

Language: Английский

Citations

0
Treatment of giant fecalith colonic obstruction in a patient with Duchenne muscular dystrophy using endoscopic injection of hydrogen peroxide: a case report and literature review DOI Creative Commons
Cheng Huang, Zhichao Gao, Yuhang Zhang

et al.

Frontiers in Medicine, Journal Year: 2024, Volume and Issue: 11

Published: Dec. 4, 2024

Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder primarily affecting cardiac and skeletal muscles, with gastrointestinal obstruction being infrequent complication.

Language: Английский

Citations

0
Digital outcome measures in Duchene muscular dystrophy: Lessons learnt from clinical trials DOI Creative Commons
Camila Gonzalez-Barral, Laurent Servais

Journal of Neuromuscular Diseases, Journal Year: 2024, Volume and Issue: unknown

Published: Dec. 8, 2024

Duchenne muscular dystrophy is a severe neuromuscular disorder characterized by progressive muscle degeneration resulting from mutations in the dystrophin gene. Digital outcome measures offer promising alternative to traditional used clinical trials. This review explores development and application of digital dystrophy, emphasizing feasibility, reliability, sensitivity, validity these measures. The stride velocity 95th centile has been validated as robust endpoint approved for use evaluation drugs treatment European Medicines Agency. Although have potential enhance efficiency accuracy trials, challenges such limited sample sizes patient compliance persist. integration artificial intelligence into data analysis progress, but further validation required before strategies can be incorporated future trial methodologies.

Language: Английский

Citations

0