Rare Diseases
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 from the FDA/CDC
FDA OKs first-ever topical gene therapy, for rare skin disease
“Because it’s safe and easy to apply directly to wounds, it doesn’t require a lot of supporting technology or specialized expertise, making...
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
IVIG shows no impact on VTE risk in dermatomyositis patients
The rate of venous thromboembolism did not increase in dermatomyositis patients treated with intravenous immunoglobulin, compared with those not...
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.
Case Reports
Deoxycholic Acid for Dercum Disease: Repurposing a Cosmetic Agent to Treat a Rare Disease
Dermatologists should consider Dercum disease when encountering a patient with numerous painful lipomas.
Conference Coverage
Pembrolizumab monotherapy effective for rare melanoma
Patients with unresectable desmoplastic melanoma experienced a response rate that was nearly double other melanomas.
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...
Conference Coverage
Childhood lupus severity linked to social determinants of health
Social determinants of health were linked to lupus disease severity in children, but it will take more than awareness among doctors to address the...