Rare Diseases
Conference Coverage
New Registry Offers Insight Into Opsoclonus-Myoclonus Syndrome
WASHINGTON – Most patients with opsoclonus myoclonus syndrome experience ataxia, among multiple other symptoms.
News
FDA approves new treatment for hereditary transthyretin-mediated amyloidosis
Inotersen inhibits the production of amyloid in patients with the rare disorder.
Conference Coverage
IgA vasculitis may be more common in adults than assumed
Daily News Podcast
Sprain an ankle, get an opioid
And beware a nasty skin souvenir from travel abroad.
Conference Coverage
Certain skin conditions signal potential overgrowth disorder
LAKE TAHOE, CALIF. – Mosaicism “can affect any tissue, anywhere, in any pattern,” expert says.
News
Bertilimumab granted orphan drug status for bullous pemphigoid
A pivotal phase 2/3 trial of bertilimumab will begin next year, according to the company.