Step 2: The Road Map to CMT Clinical Trials

Step 2 is Natural History Studies
These studies are a critical requirement for any human clinical trial. GRIN’s Natural History Studies are vital to characterizing the different manifestations of CMT and understanding the progression of the disease and loss of function over time. They allow us to identify patients for recruitment and design successful clinical trials.
In 2023, HNF has grown GRIN to over 5000+ participants with 41 CMT mutations in 60 countries, and we’ve launched a pediatric natural history study. Our latest product is the CMT Pain Management Guide.
HNF’s CMT Genie supports patients to confirm the genetic mutation of their CMT so that their symptoms and lived experiences can be correlated to their specific mutation. Since most therapeutic approaches target specific CMT subtypes, genetic testing is more important than ever.
In 2023, over 300 patients participated in the CMT Genie, with over 130 individuals testing positive for some subtype of CMT. For those whose results were negative or inconclusive, HNF continues to support our community with the next steps and additional options that will help lead to the answers we all deserve.
Watch our 4 steps here! www.hnf-cure.o...

[@suryamovies7094]

Hii MAM/SIR I'm AMAN from INDIA , BIHAR I suffering from CHARCOT MARRIE TOOTH DISEASE TYPE 2A (CMT2A). with MFN2+ gene transcription. Regarding this, I have a question whether there is any cure for it or not, if not, then how long is its treatment possible and the drug that is going to come for CMT 1A (PXT3003) can be effective in it or not... Please 🙏 reply me as well as possible.. please

[@suryamovies7094]

Hii MAM/SIR I'm AMAN from INDIA , BIHAR I suffering from CHARCOT MARRIE TOOTH DISEASE TYPE 2A (CMT2A). with MFN2+ gene transcription. Regarding this, I have a question whether there is any cure for it or not, if not, then how long is its treatment possible and the drug that is going to come for CMT 1A (PXT3003) can be effective in it or not... Please 🙏 reply me as well as possible.. please

[@suryamovies7094]

Hii MAM/SIR I'm AMAN from INDIA , BIHAR I suffering from CHARCOT MARRIE TOOTH DISEASE TYPE 2A (CMT2A). with MFN2+ gene transcription. Regarding this, I have a question whether there is any cure for it or not, if not, then how long is its treatment possible and the drug that is going to come for CMT 1A (PXT3003) can be effective in it or not... Please 🙏 reply me as well as possible.. please