dm+d
Unassigned
New Medicines
SyngilityCharcot-Marie-Tooth disease (CMT) type 1a in adults
Information
Syngility
New formulation - repurposed drug
Pharnext
Pharnext
Development and Regulatory status
Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes
Category
A low dose fixed combination of baclofen, naltrexone and sorbitol developed as a Pleodrug (a combination of off-patent drugs already approved for other indications that are unrelated clinically, but linked underlying biological networks).
An inherited sensorineural peripheral polyneuropathy first described independently by Charcot and Marie in France and Tooth in England in 1886. It is the most common inherited neuromuscular disorder affecting 10-36/100,000 individuals [2]. PXT3003 aims to lower toxic PMP22 gene over-expression in CMT1A
Charcot-Marie-Tooth disease (CMT) type 1a in adults
Oral