Rare Diseases
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 from the FDA/CDC
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
Will TP53-mutated AML respond to immunotherapy?
NATIONAL HARBOR, MD. – Patients with TP53-mutated AML were shown to have increased immune infiltration, but it isn’t clear if they will respond...
Conference Coverage
Serum test sheds light on Merkel cell carcinoma
LAS VEGAS – The rare skin cancer is linked to a subclinical virus and a serum test can offer clinicians useful insight into tumor burden.
Conference Coverage
Better overall survival with nivolumab vs. chemo for advanced ESCC
BARCELONA –Nivolumab was associated with improved overall survival and a favorable safety profile vs. chemotherapy...
From the Journals
Adverse cytogenetics trump molecular risk in NPM1-mutated AML
The findings have implications for postremission treatment decisions, including whether NPM1-mutated patients should receive allogeneic...
News
FDA approves afamelanotide for treatment of rare condition with light-induced pain
This is the first treatment approved to help patients with erythropoietic protoporphyria increase their exposure to light.
Conference Coverage
mIDH1 inhibitor ivosidenib improves progression-free survival in advanced cholangiocarcinoma
BARCELONA - The first-in-class oral inhibitor of mIDH1, significantly improved PFS for advanced cholangiocarcinoma in the global, randomized,...
From the Journals
Anakinra treatment for pediatric ‘cytokine storms’: Does one size fit all?
A retrospective cohort study saw mortality reduced if anakinra was administered early to treat sHLH/MAS. But experts caution that anakinra may not...
From the Journals
Can we eradicate malaria by 2050?
A Lancet Commission report details a series of modeled projections that could point the way.
News
FDA approves Turalio for symptomatic tenosynovial giant cell tumor
Turalio is the first therapy approved for the rare joint tumor and is available only through the Turalio Risk Evaluation and Mitigation (REMS)...