News

ReviR Therapeutics Announces First Participant Dosed in Phase 1 Study and Secures CDE Clearance for Two Orphan Indications in China, Supporting Future U.S. Expansion Strategy Milestone marks the first clinical program for Charcot-Marie-Tooth disease and Vanishing White Matter disease. SAN FRANCISCO and SHANGHAI, March 2, 2026 /PRNewswire/ -- ReviR Therapeutics, a clinical-stage biotechnology company pioneering AI-driven RNA-modulating small molecules to treat rare and genetic diseases, today announced a pivotal clinical and dual regulatory milestone: the first participant has been dosed…

Cure CMT, a patient-driven venture philanthropy organization dedicated to accelerating treatments for Charcot–Marie–Tooth (CMT) disease, today announced a strategic investment in EverTree Bio to support the advancement of the company’s therapeutic program for Charcot–Marie–Tooth disease type 1A (CMT1A). The investment reflects Cure CMT’s mission to catalyze high-impact, translational programs that can meaningfully improve outcomes for patients and families. The investment was evaluated through Cure CMT’s CURES Team, the organization’s scientific and diligence committee. The review included scientific rationale, development feasibility,…

The European CMT Federation is proud to highlight the publication of a definitive new review in Nature Reviews Disease Primers. Authored by world-leading experts—including key members of the European CMT Research Association (ECRA) Board—this document sets the standard for our understanding of CMT for the next decade. This comprehensive update moves beyond traditional knowledge, offering critical insights into: Global Epidemiology: Revised prevalence data revealing significant geographic variability and underdiagnosis. Diagnostic Innovation: The shift toward Long-read Whole Genome Sequencing to uncover…

At Cure-CMT, we're on a mission to make an immediate impact on the lives of people living with CMT. Through strategic investments and partnerships with scientists, medical experts, industry leaders, and the CMT community, we're driving innovation and pushing promising technology forward at an unprecedented pace. Since our inception, we've been executing on our mission by providing direct funding for braces and surgery so people can move with confidence, reduce pain, and regain independence. We've also made strategic philanthropic investments…