Rare Diseases
Conference Coverage
When to suspect calciphylaxis and what to do about it
Calciphylaxis is a skin condition with no generally accepted diagnostic criteria clinically, radiographically, or histologically.
Conference Coverage
Are CRMO and SAPHO syndrome one and the same?
MAUI, HAWAII – Chronic recurrent multifocal osteomyelitis is not just for kids.
News
Osilodrostat gets FDA go-ahead for Cushing’s disease in adults
The oral cortisol synthesis inhibitor is the first therapy approved by the agency to address the overproduction of cortisol.
News
Upcoming vaccine may offset surge in polio subtypes
Response to polio outbreaks involves fast-tracking a modified oral vaccine.
Conference Coverage
Deferiprone noninferior to deferoxamine for iron overload in SCD, rare anemias
An oral iron chelator granted accelerated approval for thalassemia is noninferior to deferoxamine for iron overload in patients with sickle cell...
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
Fewer people are dying from systemic sclerosis at younger ages
ATLANTA – Mortality of people with systemic sclerosis in the United States aged 44 and younger now rivals the death rate for the general...
Conference Coverage
RNA inhibitors silence two new targets in dyslipidemia
PHILADELPHIA –
Conference Coverage
Apremilast for Behçet’s oral ulcers: Benefits maintained at 64 weeks
MADRID – Reassuring results of a long-term extension of the phase 3 RELIEF trial.