Rare Diseases
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.
News
FDA OKs new drug for Fabry disease
“The totality of clinical data suggests that Elfabrio has the potential to be a long-lasting therapy.”
From the Journals
Cutaneous vasculitis curtails quality of life
Survey respondents described a generally poor health-related quality of life that was detailed as more severe with a skin-specific instrument than...
From the Journals
Study shifts burden of IgG4-related disease to women
First population-based U.S. analysis establishes “key benchmarks” for immune-mediated condition initially described in 2001.
News
FDA gives fast-track approval to new ALS drug
“The FDA’s approval of Qalsody gives me hope that people living with this rare form of ALS could experience a reduction in decline in strength,...
Latest News
Study compares noninvasive treatments of cutaneous neurofibromas
The epidermis overlying cNF is normal, “so there is no reason to use nonselective or surgical methods and destroy a perfectly good epidermis when...