Hereditary Neuropathy Foundation CMT4A therapy development project - Update Fall 2021

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Update:Year 2 of CMT4A (GDAP1) - Treatment Development & Research

 

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

 

  1. Gene Replacement Therapy (Undisclosed Biotech Company)

 

  1. HDAC6 (Undisclosed Biotech Company)

 

  1. FDA-Approved 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, send an email to registry coordinator@hnf-cure.org if you have an iPhone and want to use the mobile app. You will receive a link to enroll in the new mobile app for our clinical virtual Natural History Study!

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