New Treatment Awards

Each year, WORLDSymposia recognizes important achievements in therapy for lysosomal diseases. In 2015, several new treatments were recognized for achieving regulatory approval, and received the WORLDSymposium New Treatment Award:

Cerdelga™ (eliglustat) capsules, oral therapy for certain adult Gaucher disease type 1 patients (Genzyme, a Sanofi company)

Hunterase® (idursulfase beta) for treatment of MPS II, Hunter syndrome (Green Cross Corp.)

PROCYSBI™ (cysteamine bitartrate) oral therapy for the management of nephropathic cystinosis in adults and children 6 years of age and older (Raptor Pharmaceutical Corp.)

Vimizim® (elosulfase alfa) for treatment of patients with MPS IVA, Morquio syndrome (BioMarin Pharmaceutical Inc.)

Detailed information about this award was presented at the WORLDSymposium 2015 banquet on Thursday, February 12.