Key result
Genetic therapies, including gene replacement and antisense oligonucleotide-based gene knockdown, are currently in development as candidate treatments for congenital myopathies.
Why the study?
Congenital myopathies cause severe symptoms due to muscle weakness, but there are currently no approved drug therapies for any subtype.
Genetic therapies are emerging as potential precision medicine treatments for congenital myopathies, which currently have no approved drug therapies.
Gene therapies reach clinical use in congenital myopathies; leaves open long-term efficacy and optimal patient selection.
PURPOSE OF REVIEW: There has been an explosion of advancement in the field of genetic therapies. The first gene-based treatments are now in clinical practice, with several additional therapeutic programs in various stages of development. Novel technologies are being developed that will further advance the breadth and success of genetic medicine.Congenital myopathies are an important group of neuromuscular disorders defined by structural changes in the muscle and characterized by severe clinical symptoms caused by muscle weakness. At present, there are no approved drug therapies for any subtype of congenital myopathy.In this review, we present an overview of genetic therapies and discuss their application to congenital myopathies. RECENT FINDINGS: Several candidate therapeutics for congenital myopathies are in the development pipeline, including ones in clinical trial. These include genetic medicines such as gene replacement therapy and antisense oligonucleotide-based gene knockdown. We highlight the programs related to genetic medicine, and also discuss congenital myopathy subtypes where genetic therapy could be applied. SUMMARY: Genetic therapies are ushering in an era of precision medicine for neurological diseases. Congenital myopathies are conditions ideally suited for genetic medicine approaches, and the first such therapies will hopefully soon be reaching congenital myopathy patients.
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A 2021 study conducted a review in Congenital myopathies. Genetic therapies was evaluated. Genetic therapies, including gene replacement and antisense oligonucleotide-based gene knockdown, are currently in development as candidate treatments for congenital myopathies.
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