[HTML][HTML] Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials

Y Shimizu-Motohashi, S Miyatake… - American journal of …, 2016 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is an X-linked progressive degenerative muscle
disorder caused by the absence of dystrophin. There is no curative therapy, although …

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Development of exon skipping therapies for Duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues

A Aartsma-Rus, V Straub, R Hemmings… - Nucleic acid …, 2017 - liebertpub.com
Duchenne muscular dystrophy (DMD) is a rare, severe, progressive muscle-wasting disease
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …

[HTML][HTML] Restoring dystrophin expression in Duchenne muscular dystrophy: current status of therapeutic approaches

Y Shimizu-Motohashi, H Komaki, N Motohashi… - Journal of personalized …, 2019 - mdpi.com
Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive
muscle weakness, is caused by the absence or a decreased amount of the muscle …

Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approach

G Patterson, H Conner, M Groneman, C Blavo… - European Journal of …, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that
causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein …

Exon‐skipping therapy for Duchenne muscular dystrophy

A Nakamura, S Takeda - Neuropathology, 2009 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is a lethal muscle disorder caused by mutations in the
DMD gene for which no mutation‐targeted therapy has been available thus far. However …

[HTML][HTML] Exon-skipping in Duchenne muscular dystrophy

S Takeda, PR Clemens… - Journal of neuromuscular …, 2021 - content.iospress.com
Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically
described in the 19 th century, the genetic foundation of DMD was not discovered until more …

[HTML][HTML] Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy

A Nakamura - Journal of human genetics, 2017 - nature.com
Duchenne muscular dystrophy (DMD) is an X chromosome-linked lethal muscular disorder
with progressing muscle wasting and weakness caused by mutations in the gene encoding …

Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

[HTML][HTML] Lessons learned from discontinued clinical developments in Duchenne muscular dystrophy

T Markati, L De Waele, U Schara-Schmidt… - Frontiers in …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of
functional dystrophin protein. Patients experience progressive muscle weakness …