Rare Diseases
News from the FDA/CDC
FDA okays first-ever new drug for rare bone disorder
The drug reduced annualized heterotopic ossification volume by 54%.
Latest News
Study highlights diagnostic challenges of differentiating lichen sclerosus from vitiligo
The investigators aimed to characterize differences in LS and vitiligo based on history, physical exam, and demographic findings at the initial...
Latest News
Study evaluating in utero treatment for hypohidrotic ectodermal dysplasia seeks enrollees
Researchers are seeking to enroll mothers 18 years of age and older who are genetically confirmed carriers of the XLEHD mutation and pregnant with...
Latest News
Progress seen on five fronts for substantially improving treatment of epidermolysis bullosa
Progress on multiple fronts for control of disease or its symptoms suggests “we are on the cusp of a new era.”
Latest News
Case report describes pediatric RIME triggered by norovirus
The study author says the incidence of RIME appears to be increasing, although its true epidemiology is unknown.
Latest News
In new era of gene therapy, PCPs are ‘boots on the ground’
Pediatricians and general practitioners will be central to finding and monitoring the patients that need these treatments.
Feature
Camp Discovery: A place for children to be comfortable in their own skin
Chronic skin conditions carry a heavy psychological burden, but a special 1-week camp for youth 8-16 founded 30 years ago aims to lift that weight...
Latest News
New guidelines for MTX use in pediatric inflammatory skin disease unveiled
One of the recommendations states that liver biopsy is not indicated for routine monitoring of pediatric patients taking low-dose MTX.
News from the FDA/CDC
FDA approves ritlecitinib for ages 12 and up for alopecia areata
It is the second JAK inhibitor approved for treating alopecia areata, following approval of baricitinib (Olumiant) in June 2022 for alopecia...
From the Journals
Girls with congenital diaphragmatic hernia have lower survival rates
“Although racial and ethnic outcome disparities have been documented in CDH, disparities between males and females are not well known.”
Guidelines
Systemic JIA and AOSD are the same disease, EULAR says
New recommendations state that the ultimate goal of treatment should be drug-free remission.