Rare Diseases
News
FDA approves first treatment for advanced epithelioid sarcoma
Tazemetostat earns accelerated approval for the treatment of patients aged 16 years and older with metastatic or locally advanced epithelioid...
From the Journals
Expedited review programs not shortening drug development
While it may be hard to understand the impact of the many expedited review programs introduced at the FDA, there is still room for improvement in...
Conference Coverage
Phase 2 studies show potential of FcRn blockade in primary ITP
ORLANDO – The results support continued investigation in phase 3 trial for rozanolixizumab and efgartigimod, according to investigators.
Conference Coverage
Sutimlimab boosts hemoglobin, quality of life in cold agglutinin disease
ORLANDO – A novel antibody targeted to the complement pathway increased hemoglobin and improved quality of life in patients with cold agglutinin...
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 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...