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Webinar Recordings
…treatment and the use of physical agent modalities. She has specialty training in vision and cognitive rehabilitation, as well as neurological treatment and splinting of the upper extremity. Dancing to…
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…
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….
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…
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
…category placement of some agents. As with any treatment, the risk of neuropathy exacerbation must be weighed against expected treatment benefits and available equivalent, alternative treatments. Despite the limitations of…
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…
Coronavirus (COVID-19) and CMT
…impacts will the vaccine have on possible future gene therapy treatments or medications? Whether the vaccine will have an adverse impact on any course of treatment that you or your…