CMT Research Foundation

We've been celebrating Alan Jackson's Last Call Tour, which has donated part of every ticket sold to CMT research. We know many of you have been eager to attend his last show in Nashville. Well, now everyone will get a chance to enjoy it, even those who can't make it in person!

If you'd like to join Alan Jackson in supporting CMT research, we'd welcome your contribution: https://cmtrf.org/last-call-tour-alan-jackson/

Exciting News! "Alan Jackson: The Last Show" will air later this year on NBC and stream next day on Peacock TV. Celebrating the music, memories, and fans who have been part of the journey every step of the way! Stay tuned for date and time details, so you don’t miss this unforgettable evening.

At CMTRF, we have been watching the gene therapy regulatory landscape closely for years, and this guidance is worth examining carefully.

Our read: there is genuine reason for optimism, alongside a few open questions as the guidance moves toward finalization.

New FDA Draft Guidance Creates Opportunities for Gene Therapy Development | CMT Research Foundation Read "New FDA Draft Guidance Creates Opportunities for Gene Therapy Development", and other Research News articles from the CMT Research Foundation.

REGISTER TODAY: Our GENE THERAPIES DEMYSTIFIED webinar series continues on June 18 with Dr. Megan Waldrop's "Gene Replacement for CMT2S: Challenges in Trial Design and Recruitment"...a chance to see how these technologies get developed.

Gene Therapies Demystified

Momentum keeps building! LifeArc announced a new partnership with Elpida Therapeutics to support the development of gene therapy programmes targeting neurodegenerative conditions in children, including Charcot–Marie–Tooth disease type 4J (CMT4J). CMTRF also supports Elpida's CMT4J work, and we look forward to the impact of this partnership.

LifeArc announces partnership with Elpida Therapeutics to accelerate gene therapies for ultra-rare diseases in children - LifeArc We have announced a new partnership with Elpida Therapeutics to support the development of three gene therapy programmes targeting ultra-rare neurodegenerative conditions in children – Spastic Paraplegia type 50 (SPG50), neuronal ceroid lipofuscinosis 7 (CLN7 Batten disease) and Charcot–Marie–...