Rare Diseases
Article
Presentation of a Rare Malignancy: Leiomyosarcoma of the Prostate
Prostatic leiomyosarcoma is an aggressive malignancy with a high risk of metastasis and a poor prognosis that poses unique diagnostic and...
Case Reports
Incidentally Discovered Ochronosis Explaining Decades of Chronic Pain
Although commonly detected early in life, alkaptonuria, a rare congenital metabolic disorder, can be challenging to diagnosis and treat in older...
Conference Coverage
ADA2 is a potent new biomarker for macrophage activation syndrome
ATLANTA – The hope, first author Dr. Pui Y. Lee said, is that the finding will lead to quicker recognition and treatment of MAS.
Conference Coverage
Tofacitinib improves disease activity in patients with polyarticular-course JIA
ATLANTA – The phase 3 trial looked at flare rate out to 44 weeks after patients first met response criteria following 18 weeks of open-label...
Conference Coverage
Study delineates spectrum of Dravet syndrome phenotypes
BALTIMORE – Presentations often differ subtly from the prototypical phenotype.
Conference Coverage
Efficacy of postvenetoclax therapy may depend on prior agent exposure in CLL
ORLANDO – The multicenter study was aimed at addressing the knowledge gap around what therapy to use after venetoclax.
Conference Coverage
Myeloma patients over age 70 can benefit from auto-HC transplant
ORLANDO – Researchers said all myeloma patients should be referred a transplant center, regardless of age.
Conference Coverage
High complete response rate seen with novel CAR-T for myeloma
ORLANDO – A CAR T product targeting B-cell maturation antigen, one of several in development, was associated with a 100% overall response rate.
News
FDA approves Givlaari for treatment of acute hepatic porphyria
Prior to this approval, existing treatments provided only partial relief from porphyria attacks, according to the FDA.
Conference Coverage
RNA inhibitors silence two new targets in dyslipidemia
PHILADELPHIA –