CMT4 (GDAP) Gene Therapy Research Program

CMT4 is a rare subtype of CMT, a genetic, neurological disorder that causes damage to the peripheral nerves — tracts of nerve cell fibers that connect the brain and spinal cord to muscles and sensory organs.

CMT4 is a subtype of CMT that is inherited in an autosomal recessive pattern.

“As genetic therapies quickly become a real option for CMT patients,

this new HNF project is very exciting for patients, physicians, and researchers alike”

– Dr. Stephen Züchner, MD, PhD Professor for Human Genetics and Neurology Chair, Dr. John T. Macdonald Foundation Department of Human Genetics Co-Director, John P. Hussman Institute for Human Genomics University of Miami Miller School of Medicine and founder of The Genesis Project

Estela Lugo, CMT4 Patient
 
Previously believed to have CMT2, diagnosis corrected to CMT4.