[Gene therapies for neuromuscular diseases].
Gentherapien für neuromuskuläre Erkrankungen.
Duchenne muscular dystrophy
Gene therapy
Neuromuscular diseases
Spinal muscular atrophy
Journal
Der Nervenarzt
ISSN: 1433-0407
Titre abrégé: Nervenarzt
Pays: Germany
ID NLM: 0400773
Informations de publication
Date de publication:
Aug 2019
Aug 2019
Historique:
pubmed:
10
7
2019
medline:
20
9
2019
entrez:
10
7
2019
Statut:
ppublish
Résumé
For a long time the treatment of neuromuscular diseases was considered to be purely symptomatic. Due to new technologies in recent years novel causal forms of treatment could be developed. Gene therapies for spinal muscular atrophy, Duchenne muscular dystrophy, limb-girdle muscular dystrophy, myotubular myopathy and hereditary motor and sensory neuropathy type 1A are currently being evaluated in clinical trials. Initial preliminary results are promising and the first preparation onasemnogene abeparvovec-xioi (Zolgensma®) for the treatment of spinal muscular atrophy has recently been approved by the U.S. Food and Drug Administration (FDA). This review describes the principles of gene therapy, summarizes the interim results published so far and provides an overview of currently active or soon to be initiated gene therapy trials. Gene therapies have the potential to significantly influence the course of neuromuscular diseases. First positive intermediate results have been published and the first treatment has recently been approved in the USA. Long-term data on sustained effects and toxicity of gene therapies are not yet available. These novel treatment options will present new challenges for the healthcare systems concerning diagnosis, treatment and reimbursement.
Sections du résumé
BACKGROUND
BACKGROUND
For a long time the treatment of neuromuscular diseases was considered to be purely symptomatic. Due to new technologies in recent years novel causal forms of treatment could be developed. Gene therapies for spinal muscular atrophy, Duchenne muscular dystrophy, limb-girdle muscular dystrophy, myotubular myopathy and hereditary motor and sensory neuropathy type 1A are currently being evaluated in clinical trials. Initial preliminary results are promising and the first preparation onasemnogene abeparvovec-xioi (Zolgensma®) for the treatment of spinal muscular atrophy has recently been approved by the U.S. Food and Drug Administration (FDA).
OBJECTIVE
OBJECTIVE
This review describes the principles of gene therapy, summarizes the interim results published so far and provides an overview of currently active or soon to be initiated gene therapy trials.
CONCLUSION
CONCLUSIONS
Gene therapies have the potential to significantly influence the course of neuromuscular diseases. First positive intermediate results have been published and the first treatment has recently been approved in the USA. Long-term data on sustained effects and toxicity of gene therapies are not yet available. These novel treatment options will present new challenges for the healthcare systems concerning diagnosis, treatment and reimbursement.
Identifiants
pubmed: 31286145
doi: 10.1007/s00115-019-0761-z
pii: 10.1007/s00115-019-0761-z
doi:
Types de publication
Journal Article
Review
Langues
ger
Sous-ensembles de citation
IM
Pagination
809-816Références
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