Rare Diseases
News
Feds advance cancer moonshot with expert panel, outline of goals
Possible opportunities include vaccine development, early-detection technology, single-cell genomic analysis, immunotherapy, a focus on pediatric...
News
Guidelines emphasize testing early and often for renal impairment in multiple myeloma
News
Bone sarcomas are not just for kids
Key clinical point: Sarcomas of bone have their peak incidence in children and adolescents, but are also seen in adults. Major finding: Findings...
News
Risk factors identified for thrombosis in pediatric SLE
News
FDA approves two new hemophilia therapies
The FDA approved Idelvion for hemophilia B therapy and Kovaltry to treat hemophilia A.
Commentary
EHR Report: How Zika virus reveals the fault in our EHRs
News
Human gene editing consensus study underway
A comprehensive study of the scientific underpinnings of human gene editing technologies is underway, and the multidisciplinary committee of...
Conference Coverage
Proper hydroxyurea dose tied to better survival in sickle cell anemia
Key clinical point: Proper hydroxyurea dose in adults with sickle cell anemia was linked to higher fetal hemoglobin levels, less organ dysfunction...
News
Blocking two targets boosted fetal hemoglobin expression
New findings related to fetal globin expression could open the door to novel therapies for adults with sickle cell disease and thalassemia.
Conference Coverage
EADV: Fresh insights into Gorlin syndrome
Gorlin syndrome imposes an impressively heavy burden on affected patients.