ChallengesExperts share their insights in diagnosing and managing rare conditions
- Dasatinib Gains FDA Approval for Pediatric Ph+ Acute Lymphoblastic Leukemia
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).
- Primary Haemophagocytic Lymphohistiocytosis: Overview and New Treatment Option
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.
- 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 Lymphoma
Richard 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 Therapies
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type I (MPS I).
- Gorlin Syndrome Overview
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.
- The Current State of Rett Syndrome
Daniel Glaze, Medical Director of the Blue Bird Circle Rett Center and a Professor in the Departments of Pediatrics and Neurology at Baylor College of Medicine in Houston, discusses the current state of Rett Syndrome.
- Sanfilippo Syndrome (MPS III) Overview
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome.
- FDA Approves Romiplostim for Children With Immune Thrombocytopenia
The FDA approved romiplostim for treatment of pediatric patients with immune thrombocytopenia.
- Myelodysplastic Syndromes Overview and Personalized Prediction Model for Patients
Aziz Nazha, MD of Cleveland Clinic sat down with CheckRare to provide an overview of myelodysplastic syndromes (MDS), a group of blood disorders characterized by abnormal development of blood cells within the bone marrow.