Star Therapeutics‘ experimental therapy VGA039, developed to prevent bleeding in von Willebrand disease (VWD), has been granted both rare pediatric disease and breakthrough…
Six people with treatment-resistant immune thrombocytopenia (ITP) and underlying systemic lupus erythematosus (SLE) all achieved clinical responses to CAR T-cell immunotherapy, according to a small case series. An examination of complete responders showed signs of a more complete immune reset in the bone marrow. In contrast, partial responses were…
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A man’s severe case of treatment-resistant immune thrombotic thrombocytopenic purpura (iTTP) — which resulted in severe anemia despite more than a week spent in…
