WORLDSymposium 2015 NEW TREATMENT AWARD

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.

WORLDSymposium 2015 WELCOMED LAURIE MULDOWNEY, MD, (US FDA), AS KEYNOTE SPEAKER

Laurie Muldowney, MD, was the 2015 Keynote Speaker for WORLDSymposium™ 2015. Dr. Muldowney is Medical Officer for the Division of Gastroenterology and Inborn Error Products at CDER/FDA. Her presentation, The Importance of Natural History Studies and Patient-Focused Data in Drug Development, was at 8:00 AM on Wednesday, February 11, 2015. Dr. Muldowney also spoke at the Toolkit 2015 luncheon session on the same day.

DR. STEPHEN C. GROFT RECEIVED WORLDSymposium 2015 AWARD FOR INNOVATION AND ACCOMPLISHMENT

Stephen Groft

Each year, WORLDSymposia recognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. This year, we are pleased to present the 2015 Award for Innovation and Accomplishment in the field of lysosomal disease research and therapy to esteemed colleague Dr. Stephen C. Groft. A public servant for more than four decades and a pioneer at the Office of Rare Diseases Research, Groft is recognized worldwide as a luminary and a tireless advocate for rare diseases research. Stephen C. Groft, PharmD received the 2015 award at the WORLDSymposium 2015 opening session on February 10, 2015 at 8:15 AM.