Rare Diseases
Conference Coverage
Beloranib reduces weight in Prader-Willi syndrome, but concerns remain
Key clinical point: Beloranib produces significant weight loss in patients with Prader-Willi syndrome, but concerns have arisen about associated...
News
Free Webinar Planned for Those Interested in Referring or Applying to NIH Undiagnosed Diseases Network
News
NORD to Develop Natural History Studies With 20 Rare Disease Patient Organizations
News
Patient Assistance Programs Fill Vital Need for Those With Rare Diseases
News
$6.8 Million Awarded to Study the Biology of Rare and Undiagnosed Diseases
Conference Coverage
Study lays groundwork for refractory cutaneous lupus treatment algorithms
Key clinical point: Thalidomide, methotrexate, dapsone, and belimumab may be the best alternatives for patients whose cutaneous lupus...
News
FDA: Olanzapine can cause serious skin reaction
News
Tofacitinib an alternative treatment for refractory cutaneous dermatomyositis
Key clinical point: Tofacitinib could be an alternative treatment for refractory cutaneous dermatomyositis, and should be studied further. Major...
Conference Coverage
TSH antibody levels predict Graves relapse after thionamides
Key clinical point: Opt for radioiodine when Grave’s patients present with thyroid-stimulating hormone receptor antibody levels above 12 mU/L....
Conference Coverage
Large placebo effect seen in trial of maralixibat for primary biliary cholangitis
An international phase II trial with the investigational bile acid transporter inhibitor maralixibat chloride failed to meet its primary endpoint...
Conference Coverage
Superhigh ferritin spells trouble in macrophage activation syndrome
MAUI, HAWAII – New classification criteria for MAS now available.