The Food and Drug Administration (FDA) approved Sprycel (dasatinib) in combination with chemotherapy for the treatment of pediatric patients aged ≥1 year with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).
Carol Satler, MD Vice President of Medical Affairs in North America for Sobi, discusses the ultra rare disease primary haemophagocytic lymphohistiocytosis (HLH) and a new FDA approved treatment Emapalumab.
Ellen Ritchie, MD, Associate Professor of Clinical Medicine, Weill Cornell Medical College, an expert in the field of Myeloproliferative Neoplasms (MPNs), provides a brief understanding of what this rare blood cancer is and the role patients and their families play in actively managing their rare blood cancer. MPNs are rare, …
Richard Thomas Maziarz, MD discusses the JULIET trial, a phase-2 study of tisagenlecleucel (marketed as KYMRIAH).
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type I (MPS I).
Jean Tang, MD, PhD is co-founder of PellePharm and serves on the PellePharm board of directors. She is a practicing dermatologist and associate professor of dermatology at Stanford University.