Raredr

Hematology

For matters of the blood, and how hematology can be applied to the rare disease space, use Rare Disease Report as your primary resource.
Lexicon Receives Positive Opinion for Carcinoid Syndrome Diarrhea Treatment
Xermelo has received a positive opinion for the Marketing Authorization Application from the EMA Committee for Medicinal Products for Human Use (CHMP).
Merck KGaA and Pfizer Inc. announced that the EMA Committee for Medicinal Products for Human Use (CHMP) has recommended the approval avelumab (Bavencio).
Astellas Pharmaceuticals announced that gilteritinib was granted orphan drug designation by the FDA for use in acute myeloid leukemia (AML) patients.
Srdan Verstovsek, MD, PhD; Jamile M. Shammo, MD; and Ruben Mesa, MD, discuss the importance of a risk-based approach to therapy for polycythemia vera.
Srdan Verstovsek, MD, PhD; Jamile M. Shammo, MD; and Ruben Mesa, MD, review the triggers to begin treatment for polycythemia vera and the goals of therapy.
To raise awareness for multiple myeloma and fundraising for critical myeloma research, 6 patients with multiple myeloma, will scale Mt. Fuji.
Since 2012 Haarde has ridden over 20,000 miles through 38 states and Canada to raise more than $200,000 for Save One Life. His current trip takes him to Alaska.
Adults with relapsed or refractory ALL generally have a poor prognosis with a median survival time of 3 to 5 months.
Bayer announced they have awarded over $2 million to 16 different recipients across 8 countries as part of their Bayer Hemophilia Awards Program (BHAP).
Emicizumab prophylaxis was associated with a significantly lower rate of bleeding events among patients with hemophilia A with inhibitors.
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