An overview of recent therapeutics advances for Duchenne muscular dystrophy

JK Mah - Duchenne Muscular Dystrophy: Methods and Protocols, 2018 - Springer
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. Mutations of the DMD gene destabilize the dystrophin associated glycoprotein …

Duchenne muscular dystrophy: clinical trials and emerging tribulations

PB Shieh - Current Opinion in Neurology, 2015 - journals.lww.com
Treatment for DMD is a rapidly changing field. Some of the drugs that are currently under
investigation will receive Food and Drug Administration approval in the near future. These …

Recent developments in the management of Duchenne muscular dystrophy

M Kinali, AY Manzur, F Muntoni - Paediatrics and Child Health, 2008 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common and severe childhood muscular
dystrophy, resulting in progressive muscle weakness and wasting, disability and decreased …

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 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 …

Evolving therapeutic options for the treatment of duchenne muscular dystrophy

ES D'Ambrosio, JR Mendell - Neurotherapeutics, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …

Clinical management of Duchenne muscular dystrophy: the state of the art

S Messina, GL Vita - Neurological Sciences, 2018 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastating, progressive
neuromuscular disorder for which there is no cure. As the dystrophin gene is located on the …

Innovative therapeutic approaches for Duchenne muscular dystrophy

F Fortunato, R Rossi, MS Falzarano… - Journal of Clinical …, 2021 - mdpi.com
Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …

[PDF][PDF] State of the art advances in Duchenne muscular dystrophy

H Van Ruiten, K Bushby, M Guglieri - EMJ, 2017 - emjreviews.com
Duchenne muscular dystrophy (DMD) is a severe and fatal muscle condition affecting young
children. Without interventions, affected boys lose the ability to walk independently by the …

Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy

G Cordova, E Negroni, C Cabello-Verrugio… - Frontiers in …, 2018 - frontiersin.org
Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the
most severe due to the absence of the dystrophin protein. Typical pathological features …