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P561: REVUMENIB IN PATIENTS WITH ACUTE LEUKEMIAS: COMPASSIONATE USE PROGRAM EXPERIENCE.
P998: TRETINOIN AND CALCITRIOL ENHANCE TREATMENT REGIMENS FOR JUVENILE MYELOMONOCYTIC LEUKEMIA IN EX VIVO DRUG SENSITIVITY ASSAYS.
P985: CLL-1 IS AN ADOPTIVE IMMUNOTHERAPY TARGET IN JUVENILE MYELOMONOCYTIC LEUKEMIA.
Burden of Aging: Health Outcomes Among Adolescents and Young Adults With Sickle Cell Disease.
P-055: TRIAL IN PROGRESS: A PHASE 2, OPEN-LABEL STUDY EVALUATING THE SAFETY AND EFFICACY OF THE ERYTHROCYTE PYRUVATE KINASE ACTIVATOR ETAVOPIVAT IN PATIENTS WITH THALASSEMIA OR SICKLE CELL DISEASE.
S270: LONGER-TERM ANALYSIS OF EFFICACY OF LUSPATERCEPT VERSUS PLACEBO IN PATIENTS WITH TRANSFUSION-DEPENDENT BETA-THALASSEMIA ENROLLED IN THE BELIEVE STUDY.
PB2232: TRIAL IN PROGRESS: A PHASE 2, OPEN-LABEL STUDY EVALUATING THE SAFETY AND EFFICACY OF THE ERYTHROCYTE PYRUVATE KINASE ACTIVATOR ETAVOPIVAT IN PATIENTS WITH THALASSEMIA OR SICKLE CELL DISEASE.
P1522: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA.
P1518: LONG-TERM SAFETY RESULTS OF THE BELIEVE STUDY OF LUSPATERCEPT IN ADULTS WITH ?ETA-THALASSEMIA.
PB2314: OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL.