Gene-based therapies for neuromuscular disorders

Abstract

Neuromuscular diseases (NMD) include a broad group of medical conditions with both acquired and genetic causes. In recent years, important advances have been made in the treatment of genetically caused NMD, and most of these advances are due to the implementation of therapies aimed at gene regulation. Among these therapies, gene replacement, small interfering RNA (siRNA), and antisense antinucleotides are the most promising approaches. More importantly, some of these therapies have already gained regulatory approval or are in the final stages of approval. The review focuses on motor neuron diseases, neuropathies, and Duchenne muscular dystrophy, summarizing the most recent developments in gene-based therapies for these conditions.

Update on the diagnosis and treatment of neurological diseases

“…1 The article on gene-based therapies for neuromuscular disorders, by Zanotelli, França & Marques Jr., clearly discusses the new treatments for genetically caused neuromuscular diseases, in particular the use of therapies with gene regulation, including gene replacement, small interfering RNA (siRNA), and antisense antinucleotides. 2 The review emphasizes the new treatments for motor neuron diseases, neuropathies, and Duchenne muscular dystrophy. 2 Next, the group of neurologists with expertise in movement disorders, led by Munhoz, Tumas, Pedroso & Silveira-Moriyama, presents a valuable article about the clinical diagnosis of Parkinson's disease.…”

“…2 The review emphasizes the new treatments for motor neuron diseases, neuropathies, and Duchenne muscular dystrophy. 2 Next, the group of neurologists with expertise in movement disorders, led by Munhoz, Tumas, Pedroso & Silveira-Moriyama, presents a valuable article about the clinical diagnosis of Parkinson's disease. 3 The authors masterfully discuss the history of Parkinson's disease, the clinical criteria for diagnosis, and the auxiliary diagnostic tests, in addition to the role of non-motor and pre-motor signs and symptoms.…”