Challenges
Experts share their insights in diagnosing and managing rare conditions
FDA Approves Cabozantinib for Advanced Hepatocellular CarcinomaDespite a government shutdown, the FDA approved cabozantinib (Cabometyx) as a treatment for patients with hepatocellular carcinoma (HCC) who previously received sorafenib (Nexavar).
Morquio and Maroteaux-Lamy Syndrome (MPS IV and VI) OverviewHeather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City provides an overview of Mucopolysaccharidosis type IV and VI (MPS IV and VI). Mucopolysaccharidosis type IV is also known as Morquio syndrome, is a progressive condition that mainly affects the skeleton.
Myeloproliferative Neoplasms (MPNs)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 …
Proteus Syndrome: Overview and Emerging TherapiesBrian Schwartz, Chief Medical Officer of ArQule, discusses his company focus on Proteus syndrome, a rare condition characterized by overgrowth of the bones, skin, and other tissues.
Dasatinib Gains FDA Approval for Pediatric Ph+ Acute Lymphoblastic LeukemiaThe 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).
Primary Haemophagocytic Lymphohistiocytosis: Overview and New Treatment OptionCarol 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.
Myeloproliferative Neoplasms (MPNs)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 …
CAR-T Cell Update: Therapy Improves Outcomes for Patients With B-cell LymphomaRichard Thomas Maziarz, MD discusses the JULIET trial, a phase-2 study of tisagenlecleucel (marketed as KYMRIAH).
Mucopolysaccharidosis Type I: Overview, Diagnosis Challenges, Treatments, and Emerging TherapiesHeather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type I (MPS I).
Gorlin Syndrome OverviewJean 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.