CMT1A Research Breakthrough!

A paper published December 4th on the Journal of Clinical Investigation (JCI) website reveals an exciting potential treatment for patients with Charcot-Marie-Tooth (CMT), the progressive neuromuscular disease that affects more…

Type 1 CMT Gene Therapy Project Now Underway

…CMT (type 1 and most type 4).” “Gene therapy continues to show promise for the treatment of neuromuscular disease,” said Amanda Haidet-Phillips, Ph.D., one of the MDA’s scientific portfolio directors….

CMTA Board of Directors

…because he was eager to assist the CMTA’s mission of finding treatments and a cure for CMT. He has more than three decades of business and financial management experience from…

Voice_of_the_Patient_Report_8-19-2019

…meeting, a parallel effort to FDA’s PFDD initiative to more systematically obtain patients’ perspectives on the burden of disease and impact of current treatments or lack thereof in Hyattsville, Maryland…

New Gene Therapy Development Program for CMT2A

Philadelphia, PA – September 9, 2019 – Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system (CNS) diseases, today announced…