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Research Categories Archives

FDA to Hold Patient Listening Session for CMT2C/TRPV4 

This October, the FDA will host a Patient Listening Session for the CMT2C/TRPV4 community, organized by CMTA. During this event, the FDA will hear from people living with this form of CMT about their lives, their needs, and their hopes for new treatments.

Developing an MFN1-Targeted ASO Therapy for CMT2A

CMTA is funding researchers at La Jolla Labs, working with John Svaren, PhD, to develop an ASO treatment that increases MFN1 expression as a potential way to compensate for the damaging effects of MFN2 mutations that cause CMT2A.

CMTA Invests $110,500 in Novel ASO Medicine for CMT2A

The Charcot-Marie-Tooth Association announced a $110,500 investment partnering with La Jolla Labs to develop an antisense oligonucleotide medicine targeting CMT2A, the most common axonal form of CMT, using a novel approach to increase MFN1 expression.