This trial assessed alum-formulated glutamic acid decarboxylase, the 65-kD isoform (GAD65), a major autoantigen in type 1 diabetes. In patients with recent-onset disease; the compound did not significantly alter the loss of C peptide or improve clinical outcomes.

Guidelines suggest daily inhaled glucocorticoids in young children at high risk for asthma exacerbation. These investigators found that such treatment was not superior to the intermittent use of budesonide initiated when signs and symptoms indicated an impending asthma exacerbation.

Each year, about 225 million persons have malaria, with some 781,000 associated deaths. In a preliminary report of a phase 3 trial in African children, the RTS,S/AS01 malaria vaccine had about 50% efficacy against incident malaria and 34% efficacy against severe disease.

The introduction of a transgene into human donor T cells that can kill the cells when dimerization is induced by a pharmacologic agent has improved prospects for the exploitation of T-cell graft-versus-tumor effects while controlling graft-versus-host disease.

Ivacaftor, a potentiator of CFTR, was studied in patients with cystic fibrosis (CF) who had mutations that reduced the function of the CFTR protein. Ivacaftor significantly improved FEV₁ and reduced pulmonary exacerbations; it holds promise in the treatment of selected patients with CF.

In patients with hemophilia A in whom inhibitors of factor VIII develop, the prophylactic use of a factor VIII bypassing agent (anti-inhibitor coagulant complex) three times a week significantly reduced the risk of bleeding.