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– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –
– Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –
Key Points
Donor-derived T cells with native specificity for multiple myeloid leukemia antigens can be expanded ex vivo.
Infusion of mLSTs after HCT is well tolerated and produces antileukemia effects.
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines to treat these conditions.
The Global PNH Patient Registry is a collaborative effort between the Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD®) to study PNH.
The novel agentSY-1425 combined with azacitidine appears to be active in retinoic acid receptor alpha (RARA) superenhancer–positive newly diagnosed and relapsed or refractory
Dr. Shaffer provides an overview of the haploidentical transplant process and discusses how this treatment option might be right for some MDS patients.
Tiffany Tanaka, MD (UC San Diego Health) helps MDS patients understand the most common mutations and how genetic testing can help patients get the best possible treatment.
Session recorded as part of the Spring 2021 Virtual Patient & Family Conference.
2021 Treatment update
BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced that the U.S. Food and Drug Administration (FDA) has granted