Rare Diseases
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.
Conference Coverage
When to suspect calciphylaxis and what to do about it
Calciphylaxis is a skin condition with no generally accepted diagnostic criteria clinically, radiographically, or histologically.
News
Osilodrostat gets FDA go-ahead for Cushing’s disease in adults
The oral cortisol synthesis inhibitor is the first therapy approved by the agency to address the overproduction of cortisol.
News
Upcoming vaccine may offset surge in polio subtypes
Response to polio outbreaks involves fast-tracking a modified oral vaccine.
Conference Coverage
Esophageal stricture signals urgent treatment in kids with butterfly skin
LONDON –
Conference Coverage
Prioritize oral health in children with DEB
LONDON – While it may not be the first thing on the minds of families of children with DEB, it is important to consider oral health early to...