Duchenne muscular dystrophy: from diagnosis to therapy

MS Falzarano, C Scotton, C Passarelli, A Ferlini - Molecules, 2015 - mdpi.com
Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due
to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and …

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 …

Emerging therapies for Duchenne muscular dystrophy

T Markati, M Oskoui, MA Farrar, T Duong… - The Lancet …, 2022 - thelancet.com
Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Evolving therapeutic strategies for Duchenne muscular dystrophy: targeting downstream events

JG Tidball, M Wehling-Henricks - Pediatric Research, 2004 - nature.com
Duchenne muscular dystrophy (DMD) is a progressive, lethal, muscle wasting disease that
affects 1 of 3500 boys born worldwide. The disease results from mutation of the dystrophin …

Duchenne muscular dystrophy: drug development and regulatory considerations

DE McNeil, C Davis, D Jillapalli… - Muscle & Nerve …, 2010 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is one of the most commonly recognized
dystrophinopathies. There are no approved therapeutic options available for this disease but …

Current and emerging therapies for Duchenne muscular dystrophy

M Crone, JK Mah - Current treatment options in neurology, 2018 - Springer
Purpose of review The purpose of this review is to summarize the current and emerging
therapies for Duchenne muscular dystrophy (DMD). Recent findings Coinciding with new …

Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy

G Ricci, L Bello, F Torri, E Schirinzi, E Pegoraro… - Neurological …, 2022 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastatingly severe genetic muscle
disease characterized by childhood-onset muscle weakness, leading to loss of motor …

[HTML][HTML] Advances in gene therapy for muscular dystrophies

H Abdul-Razak, A Malerba, G Dickson - F1000Research, 2016 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy
caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre …

Advances in the treatment of Duchenne muscular dystrophy: new and emerging pharmacotherapies

AM Reinig, S Mirzaei, DJ Berlau - … : The Journal of Human …, 2017 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disease that primarily
affects young males. Patients with DMD are unable to produce dystrophin, a crucial protein …

The DMD gene and therapeutic approaches to restore dystrophin

F Fortunato, M Farnè, A Ferlini - Neuromuscular Disorders, 2021 - Elsevier
Duchenne muscular dystrophy (DMD) is a severe X-linked disease characterized by
progressive muscle weakness. It is caused by a variety of DMD gene pathogenic variations …