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).
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.
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.
The FDA approved romiplostim for treatment of pediatric patients with immune thrombocytopenia.
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.