Rare Diseases
Conference Coverage
Intravenous immunoglobulin controls dermatomyositis in phase 3 trial
Intravenous immunoglobulin was safe and effective for dermatomyositis in the first multinational, phase 3, placebo-controlled trial to evaluate...
Conference Coverage
Two treatments show early promise for hypothalamic obesity
Conference Coverage
Rituximab’s serious infection risk in ANCA-vasculitis allayed by antibiotic use
Co-use of co-trimoxazole decreased the risk for infection, which remains high in patients with vasculitis who are treated with rituximab.
News from the FDA/CDC
FDA panel narrowly backs avacopan approval
The FDA is mulling whether to approve avacopan for ANCA-associated vasculitis based largely on a single trial about which the agency staff has...
Feature
VEXAS: A novel rheumatologic, hematologic syndrome that’s making waves
Conference Coverage
Hyperphagia, anxiety eased with carbetocin in patients with Prader-Willi syndrome
Conference Coverage
Success in achondroplasia spurs testing vosoritide in more growth disorders
The quality of growth achieved with vosoritide in achondroplasia is leading to studies in more growth disorders.
News from the FDA/CDC
Tocilizumab (Actemra) scores FDA approval for systemic sclerosis–associated interstitial lung disease
The drug received FDA Priority Review and is the first biologic to be approved by the agency to reduce lung function decline in systemic sclerosis...
From the Journals
Outcomes have improved for PAH in connective tissue disease
Modern treatments and combination approaches may have contributed to overall improvements in survival in patients with pulmonary arterial...
From the Journals
Tocilizumab may improve lung function in early systemic sclerosis
Treatment with tocilizumab may improve lung function in patients with interstitial lung disease associated with systemic sclerosis.
Feature
Dan Kastner wins Crafoord Prize in Polyarthritis
Dr. Kastner received the award for identifying the mechanisms responsible for familial Mediterranean fever, tumor necrosis factor receptor–...