CMT1A Ionis Press Release

New Research Shows Promise for CMT Progressive Neuromuscular Disease Potential treatment offers hope for over 2.8 million affected worldwide Glenolden PA, December 4, 2017—A new paper published December 4th on…

Data Shows Benefit with PXT3003 in Patients with CMT1A

treatments. Pharnext has two lead products in clinical development. PXT3003 completed an international Phase III trial with encouraging topline results for the treatment of Charcot-Marie-Tooth disease type 1A (‘CMT1A’) and…

Webinar Recordings

…Specific Functional Assessments to Enable More Effective Treatment(s) and Monitoring of Progression in Patients with CMT Recorded November 5, 2015 “Specific Functional Assessments to Enable More Effective Treatment(s) and Monitoring…

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 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….

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…