Rare Diseases
News
FDA approves new interferon for polycythemia vera
A longer half-life means dosing every 2 weeks, instead of weekly.
Conference Coverage
Genotype, need for transfusion predict death in VEXAS syndrome
Among patients with a methionine-to-valine substitution in the UBA1 gene, survival was significantly worse, compared with patients with two other...
From the Journals
Genes related to osteosarcoma survival identified
They predict survival and are also potential therapy targets.
Conference Coverage
Rare hematologic malignancy may first present to a dermatologist
Blastic plasmacytoid dendritic cell neoplasm involves the skin in about 80% of cases.
Conference Coverage
Targeted therapies for vascular anomalies continue to be refined
The goal of future drug therapies is to support normal growth, “so we don’t need a maximum tolerated dose,” Dr. Denise Adams said.
Conference Coverage
Two treatments show early promise for hypothalamic obesity
Feature
VEXAS: A novel rheumatologic, hematologic syndrome that’s making waves
Clinical Insights
DART trial hits the target in angiosarcoma
Perspectives
COVID-19 vaccines and cancer patients: 4 things to know
Supplements
RARE DISEASES REPORT: Cancers
Conference Coverage
Childhood Hodgkin survivors have neurocognitive impairment