Rare Disease Report
Patients & Caregivers


The note he left behind at his house read, "If today I do not kill them, their sins will continue."
The FDA has approved Gleolan (aminolevulinic acid hydrochloride, ALA HCl) for use to visualize malignant tissue during glioma surgery.
Takeda Pharmaceutical and Seattle Genetics Inc. announced that data from a Phase 3 clinical trial evaluating Adcetris in CTCL patients were published in Lancet.
At the ASCO Meeting, Serge Verstovsek, MD of the MD Anderson Cancer Center discussed the combination of ruxolitinib and azacytidine in myelofibrosis treatment.
New data concludes that molecular responses to gilteritinib directly correlated with clinical response and improved overall survival (OS) in patients with FLT3 mutation-positive (FLT3mut+) relapsed/refractory (R/R) acute myeloid leukemia (AML).
At the 2017 ASCO Annual Meeting, Rare Disease Report sat with Daniel DeAngelo, MD, PhD of the Dana Farber Cancer Institute to discuss acute myeloid leukemia.
Serge Verstovsek, MD of the MD Anderson Cancer Center sat down with RDR to discuss myelofibrosis, thrombocytosis, and potential treatment options for both.
Gerhard Zugmaier, MD of Amgen and the University of Marburg sat down with RDR to discuss acute lymphoblastic leukemia.
Data concludes that a new DTP approach enabled identification of an initial cohort of angiosarcoma patients willing to share blood and medical records.
At ASCO, data from an open-label, expanded access study involving children with relapsed/refractory B-precursor ALL was presented.
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