Pharnext, a biopharmaceutical company, has announced new positive results from the ongoing open-label extension study of their drug PXT3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A), called the PLEO-CMT-FU trial. The trial has been conducted for a total of 6 years, and the data is consistent with a long-term treatment benefit of PXT3003 in patients with mild-to-moderate CMT1A. The results indicate that PXT3003 has a good safety profile and shows a long-term treatment effect on the Overall Neuropathy Limitation Scale (ONLS), which measures functional motor disability. A group of 117 patients with mild-to-moderate CMT1A are still receiving treatment with the high dose of PXT3003 in the trial.
THERAPEUTIC RESEARCH IN ACCELERATED DISCOVERY (TRIAD) PROGRAM REQUEST FOR PROPOSAL GUIDELINES
The Hereditary Neuropathy Foundation (HNF) would like to advance preclinical research as quickly as possible if it aligns with our key objectives.
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