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• W3171210010 endingPage "6048" @default.
• W3171210010 startingPage "6048" @default.
• W3171210010 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 other therapeutics target common pathways shared by several or all CMT types. As promising treatments reach the stage of clinical translation, optimal outcome measures, novel biomarkers and appropriate trial designs are crucial in order to facilitate successful testing and validation of novel treatments for CMT patients." @default.
• W3171210010 created "2021-06-22" @default.
• W3171210010 creator A5004243222 @default.
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• W3171210010 creator A5023384380 @default.
• W3171210010 creator A5054856326 @default.
• W3171210010 creator A5087007275 @default.
• W3171210010 date "2021-06-03" @default.
• W3171210010 modified "2023-10-16" @default.
• W3171210010 title "Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies" @default.
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