GlobalData on MSN3 个月
How a patient helped shape a treatment for a rare skin disorder
The treatment was already in Phase III trials ... her work advocating for EPP patients and co-founded the International ...
pharmaphorum11 天
Disc maps out US filing for porphyria drug bitopertin
Bitopertin has been awarded rare paediatric disease designation (RPD) by the FDA in May for the treatment of EPP. According to the American Porphyria Foundation (APF), EPP and XLP combined are the ...