Articles related to BLOOD DISORDERS

A quartet of current clinical trials involving first-in-class molecules and CRISPR gene editing show great promise. STELLAR investigates a fusion protein in PAH, CodeBreak 200 studies a first-in-class of KRAS inhibitor, EMERGENT-2 focuses on a new treatment for schizophrenia, and NCT04601051 studies a gene editing drug for one form of amyloidosis.

Memorial Sloan Kettering oncologist Raajit Rampal presents a case report, discusses the rationale for the few treatment options available, and summarizes the 5-year follow-up data for the RESPONSE and RESPONSE 2 trials.

In this first part of a three-case series, a 65-year-old man received ruxolitinib for treatment and reported feeling much better after one month, and after two months of treatment, most of his symptoms had completely resolved and his blood cell counts were maintained under control.

Can you determine what the appropriate treatment is for a 65-year-old man is diagnosed with multiple segmental pulmonary emboli?

Follow this case study of a 71-year-old man found to have significant, asymptomatic hypokalemia discovered in routine outpatient lab work. How would you manage this patient?

Parallels and distinct features among paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are discussed against the background of thrombosis, complement activation via eculizumab, and potential complement diagnostic and therapeutic avenues.