A gene therapy is within our reach for GDAP1 (CMT4A), an autosomal recessive loss of function disorder and effecting many patients like Owen.
The Future of CMT Research
The CMT&ME app has updated the research app to include a brand new medical profile.
StarWise Therapeutics & HNF Form A Strategic Alliance to Bring a NextGen HDAC6 Drug Therapy to CMT2A (MFN2) Patients
HNF has been committed to funding HDAC inhibitor research since 2014.
CMT&Me is an observational, non-interventional study, sponsored by Pharnext, to collect Real-World Evidence (RWE) from people living with CMT.
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
PARIS, December 18th, 2014 – Pharnext SAS today announced the proof of concept of its pleotherapy research and development approach based on a proprietary network pharmacology platform that identifies synergic combinations of drugs already approved for other diseases. Indeed, Pharnext’s lead pleodrug, PXT-3003, has shown positive results both in preclinical and Phase 2 clinical studies published today in the Orphanet Journal of Rare Diseases.
Research on CMT is global, and covers both laboratory and clinical studies. It is critically important to be aware of what is happening elsewhere as well as in the USA because it can have implications for what we do and fund at the HNF.
Some recent papers on Charcot-Marie-Tooth (CMT) disease go to show that we are steadily and impressively peeling back the complexity of the biology even though it is a relatively common rare disease with several thousand publications on it.