Rare Diseases
Conference Coverage
ADVOCATE: Avacopan shows renal benefits in ANCA vasculitis
A subanalysis of renal effects in the phase 3 ADVOCATE trial found that avacopan improved estimated glomerular filtration rate best among patients...
Conference Coverage
Genotype, need for transfusion predict death in VEXAS syndrome
Among patients with a methionine-to-valine substitution in the UBA1 gene, survival was significantly worse, compared with patients with two other...
Conference Coverage
Evaluations of novel approaches to treating NF-1 tumors are underway
“We need a way to inhibit the cutaneous neurofibromatosis early in life and prevent disfigurement that occurs when kids become adults.”
Feature
WHO unveils global roadmap to defeat meningitis by 2030
News from the FDA/CDC
FDA approves avacopan for rare ANCA autoimmune disease
ChemoCentryx said it plans to price its new medicine at an annual cost of $150,000-$200,000 for U.S. patients.
From the Journals
Genes related to osteosarcoma survival identified
They predict survival and are also potential therapy targets.
Conference Coverage
Several uncommon skin disorders related to internal diseases reviewed
Dermatologist discusses sarcoidosis, Rosai-Dorfman disease, Erdheim-Chester disease, and more.
News from the FDA/CDC
FDA approves intravenous immunoglobulin for dermatomyositis
Octagam 10% is the first treatment to have an indication specifically for dermatomyositis.
From the Journals
Two case reports identify Guillain-Barré variants after SARS-CoV-2 vaccination
Investigators in India and the United Kingdom describe rare presentations of Guillain-Barré syndrome marked by facial paralysis, but evidence of...
Conference Coverage
New biomarkers may predict interstitial lung disease progression in patients with systemic sclerosis
Radiographic assessment of interstitial lung disease progression and proteins from bronchoalveolar lavage may independently provide better insight...
Conference Coverage
EULAR, ACR present preliminary recommendations for rare genetic autoinflammatory diseases
Recent genetics research into several rare autoinflammatory diseases has provided enough data for EULAR and ACR to issue the first recommendations...