Rare Diseases
News
Clinical benefits persist 5 years after thymectomy for myasthenia gravis
Patients with generalized nonthymomatous myasthenia gravis who undergo thymectomy may have better long-term clinical outcomes and require less...
Daily News Podcast
Aspirin and Omega-3 fatty acids fail
Flu activity is ‘high’ in nine states.
News
FDA approves ravulizumab for treatment of paroxysmal nocturnal hemoglobinuria
The drug’s novel formulation means that patients can be treated every eight weeks, instead of every two weeks as is the case with the previously...
Daily News Podcast
Parental leave for residents
Social media can help you stay up-to-date: advice on how to use it.
News
CDC: No medical therapy can yet be recommended for acute flaccid myelitis
Updated guidelines find no solid evidence behind any treatment, all which must still be considered anecdotal.
From the Journals
AEDs strongly linked to rare serious skin reactions
The highest-risk AEDs were zonisamide, rufinamide, clorazepate, lamotrigine, carbamazepine, and phenytoin.
News
FDA approves Sympazan for Lennox-Gastaut syndrome
Patients with Lennox-Gastaut syndrome can have difficulty swallowing pills or large-volume oral suspensions, so this oral film may reduce care...
Conference Coverage
Pediatric indications appear in one-third of orphan drug approvals
WASHINGTON – Many pediatric indications involve genetic disorders.
News from the FDA/CDC
FDA approves Xyrem to treat children with narcolepsy
The central nervous system depressant was approved for the treatment of cataplexy and excessive daytime sleepiness.
Video
Technology offers new tools, challenges for rare-disease patients
WASHINGTON –
Video
Education and support enhance care for rare-disease patients
WASHINGTON –