CLINICAL PERSPECTIVES +++++

CLINICAL PERSPECTIVES Ongoing Phase 3 Study of Paltusotine in Carcinoid Syndrome Due to Neuroendocrine Tumors https://checkrare.com/ongoing-phase-3-study-of-paltusotine-in-carcinoid-syndrome-due-to-neuroendocrine-tumors/ Aman Chauhan, MD, Oncologist at the University of Miami, discusses details of a phase 3 study testing paltusotine in patients with carcinoid syndrome due to neuroendocrine tumors (NETs). Results from the ElevAATe Clinical Trial of Efdoralprin Alfa for Patients With AAT Deficiency https://checkrare.com/results-from-the-elevaate-clinical-trial-of-efdoralprin-alfa-for-patients-with-aat-deficiency/ Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the ElevAATe clinical trial of efdoralprin alfa for the treatment of patients with alpha-1 antitrypsin deficiency (AATD). Ipsen’s Current Rare Disease Therapies: Approved and In Development https://checkrare.com/ipsens-current-rare-disease-therapies-approved-and-in-development/ Christelle Huguet, PhD, Head of Research and Development at Ipsen, discusses the company’s current approved orphan drugs and those in development for rare diseases. Sophie’s Hope Foundation: A GSD1b Patient Advocacy Organization https://checkrare.com/sophies-hope-foundation-a-gsd1b-patient-advocacy-organization/ Jamas LaFreniere, Founder of Sophie’s Hope Foundation and CURE GSD1b, and Blair Stone-Schneider, Executive Director of Sophie’s Hope Foundation, discuss their patient advocacy organization and starting a natural history study for glycogen storage disease type 1b (GSD1b). Radiopharmaceutical Treatment for Neuroendocrine Tumors https://checkrare.com/radiopharmaceutical-treatment-for-neuroendocrine-tumors/ Erik Mittra, MD, PhD, Professor of Diagnostic Radiology Oregon Health and Science University, discusses neuroendocrine tumors.