Posted by Michael Wonder on 20 Aug 2020
Polaryx Therapeutics receives FDA fast track designation to PLX-200 for the treatment of patients with juvenile neuronal ceroid lipofuscinosis
20 August 2020 - Polaryx Therapeutics announced today that the U.S. FDA has granted fast track designation to PLX-200 for the treatment of juvenile neuronal ceroid lipofuscinosis patients.
Juvenile neuronal ceroid lipofuscinosis is a rare and fatal genetic lysosomal storage disorder caused by a Cln3 mutation. It is the most prevalent among the neuronal ceroid lipofuscinosis diseases and occurs in 3 out of 100,000 births.
