Search Results for: treatment

Webinar Recordings

…Rehabilitation Institute at Vanderbilt since 2008. Valery has spent the last decade specializing in treatment of adults with acquired neurological impairments. She is certified in neuro-developmental treatment and the use…

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…

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….

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-STAR Biotech & Alliance Partners

…the development of innovative therapeutics harnessing the Integrated Stress Response (ISR) for the treatment of a broad range of diseases. The company plans to demonstrate the clinical effectiveness of its…

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…

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…