Rare Diseases
News from the FDA/CDC
FDA approves intravenous immunoglobulin for dermatomyositis
Octagam 10% is the first treatment to have an indication specifically for dermatomyositis.
Case Reports
27-year-old woman • postpartum seizures • PTSD • history of depression • Dx?
► Postpartum seizures
► Posttraumatic stress disorder
► History of depression
From the Journals
Two case reports identify Guillain-Barré variants after SARS-CoV-2 vaccination
Investigators in India and the United Kingdom describe rare presentations of Guillain-Barré syndrome marked by facial paralysis, but evidence of...
From the Journals
U.S., international MIS-C studies yield disparate results
The two real-world observational studies were published online in The New England Journal of Medicine.
Conference Coverage
EULAR, ACR present preliminary recommendations for rare genetic autoinflammatory diseases
Recent genetics research into several rare autoinflammatory diseases has provided enough data for EULAR and ACR to issue the first recommendations...
Conference Coverage
Intravenous immunoglobulin controls dermatomyositis in phase 3 trial
Intravenous immunoglobulin was safe and effective for dermatomyositis in the first multinational, phase 3, placebo-controlled trial to evaluate...
Conference Coverage
Sickle cell disease: Epidemiological change in bacterial infections
Prospective study finds Salmonella to be most common agent responsible for invasive bacterial infection, and calls into question...
Conference Coverage
Two treatments show early promise for hypothalamic obesity
Conference Coverage
Hyperphagia, anxiety eased with carbetocin in patients with Prader-Willi syndrome
Conference Coverage
Success in achondroplasia spurs testing vosoritide in more growth disorders
The quality of growth achieved with vosoritide in achondroplasia is leading to studies in more growth disorders.
Feature
MIS-C follow-up proves challenging across pediatric hospitals
First came the challenge of figuring out the best treatment regimen for patients with MIS-C. Then physicians had to think about follow-up.