Rare Disease Report


Yesterday, UroGen Pharma Ltd released positive data from its interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel) for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).
The U.S. FDA has completed their Priority Review and granted full approval to avatrombopag (Doptelet) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.
Heather Landau, M.D., Assistant Attending Physician at the Memorial Sloan Kettering Cancer Center, explains AL Amyloidosis. In this video, she describes in detail the clinical definition of the disease, what organs are affected, and early presentation.
Findings from a study uncovered genetic subtypes of DLBCL with distinct genotypic, epigenetic, and clinical characteristics, providing a potential nosology for precision-medicine strategies in diffuse large B-cell lymphomas.
Last night, SELLAS Life Sciences Group, Inc. announced that the U.S. FDA has granted orphan drug designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.
Cellectar Biosciences has announced that the U.S. FDA granted orphan drug designation to CLR 131 for the treatment of rhabdomyosarcoma, a rare pediatric cancer.
New data revealed by the Tufts Center for the Study of Drug Development concludes that orphan drug development takes 18% longer than the average time required for other new drugs.
DNAtrix will report data from an ongoing Phase 1 trial of DNX-2401 in pediatric patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPG), which indicated efficacy through a prolonged survival rate. 
Results from the pivotal QuANTUM-R phase 3 study of single agent quizartinib significantly prolongs overall survival compared to chemotherapy in patients with relapsed/refractory AML with FLT3-ITD mutations.
FDA approves dual drug treatment, dabrafenib (Tafinlar) and trametinib (Mekinist), for Anaplastic Thyroid Cancer (ATC).
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