Rare Diseases
Conference Coverage
Intraventricular enzyme replacement slows CLN2 disease progression
LOS ANGELES – “Durable and persistent” benefit seen in open-label trial of cerliponase alfa in neuronal ceroid lipofuscinosis type 2 disease.
Daily News Podcast
MDedge Daily News: Which nonopioids are ripe for abuse?
And bum kidneys shouldn’t stop dabigatran reversal.
News
FDA approves burosumab to treat X-linked hypophosphatemia
Clinical trials showed normalization of phosphorus levels and radiographic improvement.
From the Journals
Longer poly-JIA inactivity not necessarily better before stopping anti-TNF therapy
Data “do not support the existence of a protective effect of longer duration of clinical inactive disease before considering stopping anti-TNF...
From the Journals
Chikungunya infection in children masquerades as SJS-TEN
Don’t misdiagnose presentations of vesiculobullous and necrotic lesions.
Feature
‘Right to try’ bill passes House
The bill passed despite opposition from a broad coalition of physician and patient groups.
From the Journals
RF-positive polyarticular JIA looks like adult RA
A genetic study reveals close ties between RF-positive polyarticular juvenile idiopathic arthritis and adult rheumatoid arthritis.