Rare Diseases
Latest News
Topical treatment for EB recommended for approval in the EU
“The benefit of Filsuvez is its ability to promote healing of EB partial thickness wounds,” the EMA said in an announcement.
Conference Coverage
Steroids counter ataxia telangiectasia
Red blood cells infused with dexamethasone sodium phosphate delayed symptom progression and improved survival.
From the Journals
JIA disease activity, disability linked to social factors
For patients with polyarticular juvenile idiopathic arthritis, levels of disease activity and disability vary, depending on family income,...
Conference Coverage
Novel drug significantly reduces tics in Tourette syndrome – without side effects
Ecocipam does not lead to weight gain, anxiety, depression, or tardive dyskinesia, compared with placebo – a factor that may lead to better...
Latest News
Early puberty cases among girls surged during pandemic
Doctors have tied the current uptick to the stress of the pandemic and lockdowns, including reduced physical activity and increased consumption of...
From the Journals
IV gentamicin improves junctional epidermolysis bullosa in children
After treatment, each of the children had increased, sustained expression of laminin 332.
Guidelines
Kawasaki disease guideline highlights rheumatology angles
“This guideline approaches Kawasaki disease from a perspective of acting as an adjunct to the already existing and excellent...
Guidelines
New JIA guidelines emphasize earlier DMARD use
Advances in understanding and treating systemic JIA have led practitioners away from steroids and toward using conventional synthetic and biologic...
Guidelines
New MIS-C guidance addresses diagnostic challenges, cardiac care
New updates in this third version of the American College of Rheumatology’s guidance include a recommendation for dual therapy with IVIg and...
Commentary
Growth hormone therapy for certain children may help them reach their potential
When should parents consider growth hormone therapy? In what cases is it warranted for children? Dr. Jessica Sparks Lilley discusses the need for...
Feature
Cystic fibrosis in retreat, but still unbeaten