CMTA’s 40 Under 40

treatment of SORD-CMT is currently active, only two years after the identification of the SORD gene, which, if successful, may represent the first available treatment for patients with CMT. Read…

CMTA and Pharnext Enter Biomarker Research Collaboration

…is currently no treatment available.” About Pharnext Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for orphan and common neurodegenerative diseases that currently lack curative and/or disease-modifying treatments….

Sorbitol Dehydrogenase (SORD) Deficiency

What is CMT-SORD? CMT-SORD, also known as Sorbitol Dehydrogenase Deficiency, is a recently discovered, unique type of Charcot-Marie-Tooth disease (CMT). Identified in May 2020 with funding from CMTA, this type…

Medication Study

…lithium, and pyridoxine. Overall, Weimer and Podwall conclude that treatment with vincristine poses an “unacceptable risk to patients with known or possible CMT1A.” They further conclude that the use of…