Search Results for: treatment

Initiation of Registrational Phase 2/3 Study of AT-007 in SORD Deficiency

…Deficiency patients, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment. The range of reduction from baseline in patients was 54%-75%. AT-007 was safe…

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….

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 Announces Strategic Partnership with Acceleron to Advance CMT Treatment Options

…treatments, and ultimately, a cure for CMT. Strategic alliances with corporate partners like Acceleron support innovative drug development with a goal to rapidly deliver therapies to CMT patients. “We are…

MDA and CMTA Partner to Advance Treatments and Care for Charcot-Marie-Tooth Disease

…is one of the neuromuscular diseases MDA fights as an umbrella organization with a big picture perspective on finding treatments and cures for kids and adults whose weakening physical strength…

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….

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…

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…

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…

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…