Current and emerging treatment strategies for Duchenne muscular dystrophy

JK Mah - Neuropsychiatric disease and treatment, 2016 - Taylor & Francis
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …

Update on the management of Duchenne muscular dystrophy

AY Manzur, M Kinali, F Muntoni - Archives of disease in childhood, 2008 - adc.bmj.com
Duchenne muscular dystrophy (DMD) is familiar to paediatricians as the most common
childhood muscular dystrophy and leads to severe disability and early death in the late …

Recent advances in the management of Duchenne muscular dystrophy

EM Strehle, V Straub - Archives of disease in childhood, 2015 - adc.bmj.com
Duchenne muscular dystrophy (DMD) is the commonest inherited neuromuscular disorder of
childhood and mainly affects males. Over the course of the last century, the average life …

Duchenne muscular dystrophy: a practice update

R Suthar, N Sankhyan - The Indian Journal of Pediatrics, 2018 - Springer
Abstract Duchenne Muscular Dystrophy (DMD) is an X-linked recessive disorder caused by
a deficient or defective synthesis of dystrophin protein. DMD is the most common form of …

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 …

Review of Duchenne muscular dystrophy (DMD) for the pediatricians in the community

S Verma, Y Anziska, J Cracco - Clinical pediatrics, 2010 - journals.sagepub.com
Duchenne muscular dystrophy (DMD), an inherited X-linked recessive disorder, is
characterized by progressive symmetric muscle weakness and gait disturbance, with onset …

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 …

Impending therapies for Duchenne muscular dystrophy

TA Partridge - Current opinion in neurology, 2011 - journals.lww.com
Although genetic and cell-mediated approaches are currently showing genuine promise in
preclinical and clinical trials, there remains considerable interest in the development of …

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 …

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 …