The Global Commission to End the Diagnostic Odyssey for Children, co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, is a multidisciplinary group of patient advocates, physicians and other experts working together to help solve the complex challenges impacting the rare disease community.
Approval allows Onivyde (plus oxaliplatin, fluorouracil, and leucovorin) as first-line therapy.
Tyler Sandahl, MD, Clinical Pharmacist at the Mayo Clinic, discusses the use of Tocilizumab in multiple myeloma patients.
Cat Lutz, Ph.D., M.B.A. discusses how Jackson Laboratory uses mice in testing for human disease.Cat Lutz, Ph.D., M.B.A. Vice President of the Rare Disease Translational…
Monica Gadelha, DR, PhD, University of Rio De Janeiro, discusses her acromegaly research.
Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory…
Robin Kate Kelley, MD, Professor of Clinical Medicine in the Division of Hematology/Oncology, University of California, San Francisco discusses treatment options for biliary tract cancers and…
Christine Eng, Chief Medical Officer at Baylor Genetics, discusses the information her company presented at various conferences.
Ozlem Goker-Alpan, MD, Founder and CMO of LDRTC, discusses the importance of newborn screening for lysosomal disorders.
