Search results for: treatment
Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)
About this Study First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy...
Anatomic Inc.
Anatomic provides high-quality human iPSC-derived cells and specialized differentiation technologies to support CMT research and...
Seven Reasons to Attend the CMTA Patient & Research Summit in October
Need a reason to go to the CMTA Patient & Research Summit in San Francisco...
CMTA Advances Drug Repurposing with an Additional $225,720 Investment in CMT1B and CMT1E Research
CMTA is investing $225,720 to expand drug repurposing research into CMT1B and CMT1E, building on ongoing CMT1A research targeting shared disease mechanisms.
New CMTA-Funded Research to Test if an Existing Drug Can Help People with CMT1B and CMT1E
CMT1B and CMT1E can take away strength, feeling, and balance from damage to the nerve...
New Clinical Trial Recommendations for CMT Help Speed Therapies to Patients
CMTA is leading efforts to improve CMT clinical trials through new recommendations that help create a clearer path for developing and approving new therapies.
CMTA-STAR Alliance Partner Vanda Pharmaceuticals Receives FDA Rare Pediatric Disease Designation for CMT2S Therapy Candidate
CMTA Strategy to Accelerate Research (CMTA-STAR) Alliance Partner Vanda Pharmaceuticals has announced that the U.S. Food...
Is Spinal Fluid the Way to Gene Therapy in CMT? What a Decade of Research Reveals
Most kinds of CMT come from a problem in a single gene. Repairing that faulty...
CMTA Leading the Way in CMT1A
CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments.
CMTA research takes the stage at PNS
PNS 2026 showcased CMTA's role in advancing CMT research across the translational pipeline, from biomarkers and natural history studies to clinical trials, therapeutic development, and the scientific collaborations driving progress.
