Rare Diseases
News
Folic acid fortification leads to bigger drops in neural tube defects
Key clinical point: Women of childbearing age should take 400 mcg of folic acid daily if they might become pregnant. Women with a previous neural...
News
Long-acting pasireotide approved for acromegaly
News
FDA approves newborn screening test for SCID
Conference Coverage
Autism sibling studies beginning to yield data
News
Economic cost of autism disorders is considerable
Key clinical point: Interventions are needed for patients with autism spectrum disorders that might "reduce later high expenditures by changing...
News
Gene panel identifies residual neuroblastoma metastases
Major finding: In a multivariate analysis, patients negative for minimal residual disease had about a fourfold increased rate of progression-free...
Conference Coverage
Resurrected gemtuzumab shows benefit against pediatric AML
Major finding: Three-year event-free survival rates were 53% for children and young adults treated with gemtuzumab ozogamicin added to a standard...