Rare Disease Report


To date, OPTIMISMM is the only phase 3 study in early RRMM to report a significantly and clinically meaningful PFS improvement in LEN-exposed patients.
Prospective genomics can inform subsequent clinical trial analysis aiming at matching outcome with tumor genotyping.
Saby George, MD, FACP, and Dena Battle, president of nonprofit patient advocacy group, KCCure, discussapproval of Opdivo (nivolumab) and Yervoy (ipilimumab) therpay combination for kidney cancer. 
The US FDA has granted Astellas Pharma Inc priority review for gilteritinib for refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. 
In an article recently published by the Institute of Cancer Research, genetic testing was indicated to result in life-extending treatment for incurable pediatric brain tumors. 
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.
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