About CMT4A / GDAP1
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
Year #2 of CMT4A (GDAP1) Research & Treatments
As we enter our second year of our CMT4A (GDAP1) Gene Therapy Initiative, HNF is making progress and expanding its aggressive mission of developing potential treatments and a cure for this unique type of CMT.
“We’ve made swift progress with the success of human-derived cellular models by Dr. Mario Saporta at the University of Miami. We now have differentiated motor neuron cells from GDAP1 patients and we are ready to test multiple potential treatments. At HNF, we understand that the wisest approach is to not put all of our eggs in one basket, so in addition to the Gene Therapy approach, we’ve added 2 NEW promising pathways toward treatment development.” – Allison Moore
Three Promising Pathways for CMT4A (GDAP1) Treatment Development
- Gene Replacement Therapy (Undisclosed Biotech Company)
- HDAC6 (Undisclosed Biotech Company)
- FDA-Approved Function Drug Screening (Rarebase)
*NEW Mobile App – HNF is thrilled to be launching a new mobile app that will modernize CMT clinical trial development via AI and help to connect more people living with CMT to clinical trials and expert care. It will also help characterize the different types of CMT which is critical for research.
Next Steps: We encourage all people living with CMT4A to join our registry, GRIN. Be sure to complete our Core Natural History study – including genetic report & nerve conduction test uploads (if available)!
Next, if you have an iPhone and want to participate in this important study, send an email to [email protected]. You will receive a link to download the new mobile app for our clinical virtual Natural History Study.