- Let’s Beat CMT – 2024 CMT Awareness Month#LetsBeatCMT is a social media initiative during CMT Awareness Month to encourage the Charcot-Marie-Tooth disease community to share their CMT experiences throughout September. Post your videos and photos on social media using #LetsBeatCMT to educate the ...Read more ...
- CMTA-STAR Alliance Partner Actio Biosciences Receives FDA Orphan Drug Designation and Rare Pediatric Disease Designation for Their Treatment in Development for CMT2CThe Charcot-Marie-Tooth Association (CMTA) is pleased to share exciting news from our CMTA-STAR Alliance Partner, Actio Biosciences. The company has received both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) designation from the ...Read more ...
- CMTA Alliance Partner Applied Therapeutics Plans to Submit an NDA for CMT-SORD, a Potential First Approval for the Treatment of any type of CMTThe Charcot-Marie-Tooth Association (CMTA) is pleased to share exciting news from our CMTA-STAR Alliance Partner, Applied Therapeutics. The company plans to submit a New Drug Application (NDA) to the FDA for govorestat to treat CMT-SORD ...Read more ...
- Gene Replacement Therapy in CMT4 SubtypesThe Charcot-Marie-Tooth Association (CMTA) has announced a $240K investment in a groundbreaking gene replacement therapy for CMT4A, with potential applications for other types of CMT. This critical project is led by Xin Chen, MD, PhD, ...Read more ...
- Nanoparticles for CMT1A, CMT1B, and CMTX1: A New Frontier in Gene TherapyIn a groundbreaking three-year joint project between the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA), researchers led by Alexia Kagiava, PhD, at the Cyprus Institute of Neurology and Genetics are pioneering the use ...Read more ...
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