Rare Disease Report

ASH 2016

Thomas Winkler, M.D., staff clinician at NHLBI / NIH talks about the use of eltrombopag to possibly treat diamond blackfan anemia.
At the 58th ASH meeting in San Diego, CA, we spoke with Dr. Marina Cavazzana PhD about the latest data on bluebird bio's gene therapy to treat β-thalassemia and sickle cell disease.
David Miklos, MD of Stanford University talks about the use of ibrutinib in chronic graft versus host disease.
Saad Usmani, MD, FACP, of the Levine Cancer Institute talks about very impressive data showing the efficacy of daratumumab, lenalidomide, and dexamethasone to treat relapsed or refractory multiple myeloma patients.
Pushkal Garg, MD, of Alnylam Pharmaceuticals discusses the recent data on the efficacy and safety of givosiran to treat hepatic porphyrias.
David Kuter, MD of Massachusetts General Hospital talks about the global Gaucher earlier diagnosis consensus (GED-C) initiative that is being developed to help clinicians diagnose many of the Gaucher disease patients that may not be getting properly diagnosed because their symptoms are not as severe.
David Kuter, MD, of Massachusetts General Hospital provides an overview of Gaucher disease and how it is currently managed.
Pushkal Garg, MD, of Alnylam Pharmaceuticals discusses the recent data on the efficacy and safety of fitusiran to treat patients with hemophilia.
Jeffrey Jones, MD, MPH talks about a recent phase 1b study involving the use of obinutuzmab, ibritinib, and venetoclax to treat relapsed/refractory chronic lymphocytic leukemia (CLL).
Brendan Weiss, MD of the University of Pennsylvania talks about the need for patients with AL Amyloidosis to receive chemotherapy.
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