[Molecular therapies: present and future in neuromuscular diseases].

ABSTRACT:

Background

The possibilities in the field of molecular therapies of neuromuscular diseases have rapidly developed in recent years. First compounds are already available in clinical practice and numerous other substances are in advanced phases of clinical trials. This article gives an exemplary overview of the current state of clinical research in molecular therapies of neuromuscular diseases. It also gives a view into the near future of the clinical application, including the challenges.

Discussion

Using Duchenne muscular dystrophy (DMD) and myotubular myopathy as examples, the principles of gene addition in monogenetic skeletal muscle diseases, which are already manifested in childhood are described. In addition to initial successes, the challenges and setbacks hindering th

SUBMITTER: Ziegler A 

PROVIDER: S-EPMC10204661 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature