Retrophin signs research agreement with St Jude Children’s Hospital
Under the agreement, Retrophin will supply funds and a library of replacement therapy compounds to St Jude to test for potential use in the treatment of a rare
ReviR Therapeutics has received a rare paediatric disease designation (RPDD) from the US Food and Drug Administration (FDA) for RTX-117, its investigational oral small-molecule therapy for Charcot-Marie-Tooth disease (CMT).
The expanded alliance comprises three components co-development of present and future formulations of migalastat HCl for Fabry disease, commercialisation arrangements for all future Fabry products and increased GSK