Rare Diseases
News from the FDA/CDC
FDA adds polyarticular-course JIA to approved indications for tofacitinib
The approval is the fourth indication in the United States for tofacitinib after approvals for treatment of adults with rheumatoid arthritis,...
News
COVID-19 linked to development of myasthenia gravis
Italian researchers report three cases of myasthenia gravis onset after COVID-19 infection.
Photo Rounds
Large, painful facial cysts
News
Terlipressin squeaks by FDA review for hepatorenal syndrome 1
The FDA advisory committee’s 8-7 vote favoring approval of terlipressin for hepatorenal syndrome type 1 reflected concerns and misgivings.
News
FDA approves first oral somatostatin analog for acromegaly
The oral drug octreotide offers an alternative to long-term injections for the rare chronic disease.
News
FDA approves new treatment for Dravet syndrome
Children treated with fenfluramine experienced significantly greater reductions in the frequency of convulsive seizures than did their peers who...
News
First reported U.S. case of COVID-19 linked to Guillain-Barré syndrome
The unique clinical features in the U.S. case are urinary retention secondary to dysautonomia and ocular symptoms of...
News
Two rare neurologic conditions linked to COVID-19
Two variants of Guillain-Barré syndrome—Miller Fisher syndrome and polyneuritis cranialis—have been linked with COVID-19 infection.
Conference Coverage
A case of neutrophilic eccrine hidradenitis attributed to HIV treatment
The most common causes of NEH are hematologic malignancies and some of the chemotherapeutic agents used in treating them.
News
FDA approves Koselugo for pediatric neurofibromatosis treatment
News
First case of COVID-19 presenting as Guillain-Barré reported
A case report from China suggests a possible association between GBS and SARS-CoV-2 infection.