by Peter Ciszewski | Jan 2, 2019
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...
by Peter Ciszewski | Jan 1, 2019
By Peter Ciszewski; December 23, 2018 Richard Thomas Maziarz, MD discusses the JULIET trial, a phase-2 study of tisagenlecleucel (marketed as KYMRIAH). In this study an international team of researchers evaluated 93 patients with relapsed or refractory diffuse...
by Peter Ciszewski | Dec 30, 2018
Dr. Jonathan Peled of Memorial Sloan Kettering Cancer Center discusses his study which looked at whether pre-HCT microbiota configuration may be an important determinant of post-transplantation outcomes. The study reported from a multicenter analysis conducted...
by Peter Ciszewski | Dec 29, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type I (MPS I). This condition was once divided into three separate syndromes: Hurler syndrome (MPS I-H), Hurler-Scheie syndrome (MPS...
by Peter Ciszewski | Dec 28, 2018
Rami Levin, President of North America for Swedish Orphan Biovitrum (SOBI) discusses his company’s recent FDA approval, Sobi’s commitment to the rare disease community, and how Sobi is growing its presence in North America. We sat down with Rami Levin at...
