[Gene therapies for neuromuscular diseases].

CONCLUSION: 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. PMID: 31286145 [PubMed - as supplied by publisher]

https://www.ncbi.nlm.nih.gov/pubmed/31286145?dopt=Abstract

Source: Der Nervenarzt - July 7, 2019 Category: Neurology Authors: Saffari A, Weiler M, Hoffmann GF, Ziegler A Tags: Nervenarzt Source Type: research