Search Results for: treatment

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….

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’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…

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…

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…

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…

Medications and Charcot-Marie-Tooth Disease

There currently are no available treatments that act to slow the progression of Charcot-Marie-Tooth disease. There are promising CMT research studies that indicate that this may change in the near…