Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies.

ABSTRACT: Inherited neuropathies known as Charcot-Marie-Tooth (CMT) disease are genetically heterogeneous disorders affecting the peripheral nerves, causing significant and slowly progressive disability over the lifespan. The discovery of their diverse molecular genetic mechanisms over the past three decades has provided the basis for developing a wide range of therapeutics, leading to an exciting era of finding treatments for this, until now, incurable group of diseases. Many treatment approaches, including gene silencing and gene replacement therapies, as well as small molecule treatments are currently in preclinical testing while several have also reached clinical trial stage. Some of the treatment approaches are disease-specific targeted to the unique disease mechanism of each CMT form, while ot

SUBMITTER: Stavrou M 

PROVIDER: S-EPMC8199910 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature