Rare Diseases
Latest News
Woman with transplanted uterus gives birth to boy
It’s the first time that a baby has been born to a woman with a transplanted uterus outside of a clinical trial.
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.”
Latest News
Scientists discover variants, therapy for disabling pansclerotic morphea
The JAK inhibitor ruxolitinib dramatically improved wound healing and rashes in patients with the very rare disorder.
News from the FDA/CDC
FDA approves Yuflyma as ninth adalimumab biosimilar
The manufacturer Celltrion is also seeking an interchangeability designation from the FDA, tentatively expected in the fourth quarter of 2024.
News from the FDA/CDC
FDA approves autoinjector pen for Humira biosimilar, Cyltezo
The drug will be commercially available on July 1, 2023.