Search Results for: treatment

CMTA’s 40 Under 40

…a pivotal role in driving progress towards effective treatments and, ultimately, a more promising future for the CMT community. Trained by their elders, these young individuals are pushing the boundaries…

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…

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…

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…

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…

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…

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…

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…