Rare Diseases
News
FDA approves Otezla for treatment of Behçet’s-associated oral ulcers
The approval is based on the results of the randomized, placebo-controlled, double-blind, phase 3 RELIEF trial.
From the Journals
POEM outperforms pneumatic dilation in randomized achalasia trial
Higher success rate, but also a higher rate of reflux esophagitis, seen in patients with previously untreated achalasia.
Conference Coverage
Ebola outbreak: WHO/OCHA call for more aid, better security
Security, public trust, and a lack of resources are significant barriers to countering the current Ebola epidemic in the Democratic Republic of...
From the Journals
HCC surveillance after anti-HCV therapy cost effective only for patients with cirrhosis
Surveillance twice a year of all patients with F3 or F4 fibrosis had a less than 1% chance of remaining under a $50,000 per QALY willingness-to-...
Conference Coverage
Rozanolixizumab may offer new treatment paradigm for ITP
AMSTERDAM – “Data from this study indicate that we can achieve effective increases in platelet levels, we can observe decreasing IgG levels, and...
From the Journals
Budesonide tablets considerably outperformed placebo for active EoE
After 6 weeks, 34 of 59 patients (58%) receiving budesonide orodispersible tablets had achieved complete remission.
Latest News
New tickborne virus emerges in China
A previously unknown RNA segmented virus was associated with a febrile illness that spread to 86 patients.
Conference Coverage
Report card may foretell achalasia surgery outcomes
BALTIMORE – A report card that evaluates multiple variables after myotomy for achalasia may provide a more accurate picture of outcomes than the...
Conference Coverage
Is pro soccer a risk factor for ALS?
A higher risk of ALS and an earlier age of onset was found among professional soccer players.
Conference Coverage
Use of levoketoconazole improved several clinical features of Cushing’s disease
LOS ANGELES – Patients’ quality of life scores also grew.
Conference Coverage
Three-drug regimen shows promise for refractory primary biliary cholangitis
VIENNA – Combining ursodeoxycholic acid, obeticholic acid, and bezafibrate showed efficacy and tolerability for the toughest cases.