Rare Diseases
From the Journals
Sjögren’s syndrome most common extrahepatic PBC manifestation
Up to 73% of PBC patients also have Sjögren’s syndrome.
News
FDA approves Rebinyn for hemophilia B treatment
Rebinyn is intended to control and treat bleeding episodes, as well as provide perioperative management of bleeding.
Conference Coverage
Nusinersen for early spinal muscular atrophy: Final findings beat interim results
BOSTON – But the drug costs $750,000 a year, and questions remain about its use.
Conference Coverage
Treatment challenges for lichen planopilaris
SYDNEY – Just over half of patients with lichen planopilaris in a medical record review responded to first-line therapies, but response rates...
News from the FDA/CDC
New device that treats esophageal atresia in infants has been authorized
The FDA has authorized an esophageal atresia treatment device.
Conference Coverage
Open-capsule technique improves AIE
CHICAGO – Open-capsule budesonide reduced or eliminated chronic diarrhea for patients with adult autoimmune enteropathy.
From the Journals
Mepolizumab proves effective for eosinophilic granulomatosis with polyangiitis
The anti–interleukin-5 monoclonal antibody beat placebo for total accrued weeks of remission and remission after 36 and 48 weeks of treatment.
From the Journals
High allele level linked to lamotrigine-induced SCAR
Could higher frequency of alleles associated with drug-induced severe cutaneous adverse reactions be specific to ethnicity?