![]() Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (cald) Longterm safety outcomes include the risks for bleeding, thrombosis, infections, major adverse cardiac events, secondary malignancies, and survival. ![]() The co-primary efficacy endpoints are the proportion of patients achieving =35% spleen volume reduction as measured by magnetic resonance imaging or computed tomography imaging and the proportion of patients achieving a =50% reduction in modified total symptom score (mTSS) from baseline through 24 weeks of therapy. PMR 4154-1: Conduct a randomized, controlled trial to verify and describe the clinical benefit of Vonjo in adults with intermediate-1, intermediate-2 or high-risk myelofibrosis (MF) with platelet counts of less than 50 x 109/L. For the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 x 109/L
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