Search Results for: treatment

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…

What’s New in CMT Research and Care? A 2025 Update Published by CMTA-STAR Advisory Board Member Davide Pareyson, MD

…proper care and treatment planning. Visit CMTA’s Diagnosing CMT page to learn more. Comprehensive Care: Managing CMT Today While there are no FDA-approved treatments for CMT, CMT specialists continue refining…

Applied Therapeutics Announces Positive Sorbitol Reduction Data From the Ongoing Phase 3 INSPIRE Trial in Sorbitol Dehydrogenase (SORD) Deficiency

…a pre-specified interim analysis of the ongoing Phase 3 INSPIRE trial, AT-007 reduced sorbitol levels by a mean of approximately 52% (or approximately 16,000ng/ml) over 90 days of treatment (p…

CMTA-STAR Partner Applied Therapeutics Presents 24-Month CMT-SORD Data at PNS 2025

…Annual Meeting of the Peripheral Nerve Society (PNS). The findings highlight encouraging signs of sustained clinical benefit and reflect continued momentum in the effort to advance treatments for all forms…

Frequently Asked Questions: Ionis/CMTA 1A Research Announcement

…measures, there was an overall improvement that was observed in both animal models with ASO treatment. Another important part of this study was to evaluate ASO treated mice and rats…

Research in Charcot–Marie–Tooth Disease Type 1A (CMT1A)

…over $200,000 invested, we’re pioneering methods to evaluate potential treatments targeting myelin defects in CMT1A. Join us as we revolutionize CMT understanding and treatment, contributing to a larger framework of…

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…

Toxic Medications List For Patients with Charcot-Marie-Tooth Disease

…ill and is offered any drug treatment by their physician, concern may arise about possible drug-related side effects, and these side effects may target the peripheral nerves. It is reasonable…

Your Participation Matters: Join the CMT2S Research Study

CMT2S: A Potential New Treatment on the Horizon Charcot-Marie-Tooth Disease Type 2S (CMT2S) is a severe axonal type of CMT caused by certain autosomal recessive mutations of the IGHMBP2 gene….