Advances in Treatments in Muscular Dystrophies and Motor Neuron Disorders
| Autor: | Bhaskar Roy, Robert C. Griggs |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
Drug
Genetic enhancement media_common.quotation_subject Duchenne muscular dystrophy Disease Bioinformatics Muscular Dystrophies 03 medical and health sciences 0302 clinical medicine medicine Humans 030212 general & internal medicine Motor Neuron Disease Amyotrophic lateral sclerosis media_common business.industry Genetic Therapy Spinal muscular atrophy Motor neuron medicine.disease Clinical trial Neuroprotective Agents medicine.anatomical_structure Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | Neurologic Clinics. 39:87-112 |
| ISSN: | 0733-8619 |
| DOI: | 10.1016/j.ncl.2020.09.005 |
| Popis: | Increased understanding of disease pathophysiology and advances in gene therapies and drug technologies are revolutionizing treatment of muscular dystrophies and motor neuron disorders (MNDs). New drugs have been approved for Duchenne muscular dystrophy, spinal muscular atrophy, and amyotrophic lateral sclerosis. For other diseases, new targets have been identified, and new therapies are in clinical trials. The impact of such therapies will be fully understood only in the next decades. Cost burden and accessibility are major challenges in the wide application of new drugs. This article reviews advances in gene therapies, newly approved drugs, and therapeutic promises in muscular dystrophies and MNDs. |
| Databáze: | OpenAIRE |
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