Posted by Michael Wonder on 15 Feb 2021
Polaryx Therapeutics receives both rare paediatric disease and orphan drug designations for the treatment of Krabbe disease with PLX-300
10 February 2021 - Polaryx Therapeutics announced today that it has received both rare paediatric disease and orphan drug designations for the treatment of Krabbe disease (a.k.a. Globoid Cell Leukodystrophy) with PLX-300 from the U.S. FDA.
Krabbe disease is a rare, heritable metabolic genetic disorder that affects approximately one in 100,000 individuals in the United States.
