Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases
Open Access
- 16 September 2020
- journal article
- editorial
- Published by MDPI AG in Journal of Personalized Medicine
- Vol. 10 (3), 129
- https://doi.org/10.3390/jpm10030129
Abstract
With the development of novel targeted therapies, including exon skipping/inclusion and gene replacement therapy, the field of neuromuscular diseases has drastically changed in the last several years. Until 2016, there had been no FDA-approved drugs to treat Duchenne muscular dystrophy (DMD), the most common muscular dystrophy. However, several new personalized therapies, including antisense oligonucleotides eteplirsen for DMD exon 51 skipping and golodirsen and viltolarsen for DMD exon 53 skipping, have been approved in the last 4 years. We are witnessing the start of a therapeutic revolution in neuromuscular diseases. However, the studies also made clear that these therapies are still far from a cure. Personalized genetic medicine for neuromuscular diseases faces several key challenges, including the difficulty of obtaining appropriate cell and animal models and limited its applicability. This Special Issue “Molecular Diagnosis and Novel Therapies for Neuromuscular/Musculoskeletal Diseases” highlights key areas of research progress that improve our understanding and the therapeutic outcomes of neuromuscular diseases in the personalized medicine era.This publication has 22 references indexed in Scilit:
- Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular DystrophyJournal of Personalized Medicine, 2018
- Eteplirsen in the treatment of Duchenne muscular dystrophyDrug Design, Development and Therapy, 2017
- Current Translational Research and Murine Models For Duchenne Muscular DystrophyJournal of Neuromuscular Diseases, 2016
- Dystrophin-deficient large animal models: translational research and exon skipping2015
- Therapeutic Development in Amyotrophic Lateral SclerosisClinical Therapeutics, 2015
- Animal models for metabolic, neuromuscular and ophthalmological rare diseasesNature Reviews Drug Discovery, 2013
- Genetics of neuromuscular disordersCritical Reviews in Clinical Laboratory Sciences, 2012
- Animal Models for Genetic Neuromuscular DiseasesJournal of Molecular Neuroscience, 2008
- Animal Models of Neuromuscular Diseases: Pathophysiology and Implications for RehabilitationPhysical Medicine and Rehabilitation Clinics of North America, 1998
- Population frequencies of inherited neuromuscular diseases—A world surveyNeuromuscular Disorders, 1991