Rare Diseases
News from the FDA/CDC
Ustekinumab becomes second biologic approved for PsA in kids
The approval is based on an extrapolation of data from adult trials in active psoriatic arthritis and adult and pediatric studies in moderate to...
Case Letter
Peristomal Pyoderma Gangrenosum at an Ileostomy Site
A pyoderma gangrenosum subtype occurs in close proximity to an abdominal stoma.
Latest News
Topical gene therapy for dystrophic epidermolysis bullosa shows promise
B-VEC was well tolerated with no drug-related serious adverse events or discontinuations due to treatment.
Case Letter
Focal Palmoplantar Keratoderma and Gingival Keratosis Caused by a KRT16 Mutation
Focal palmoplantar keratoderma and gingival keratosis (FPGK) is a rare autosomal-dominant syndrome featuring focal, pressure-related, painful...
Latest News
Topical gel for epidermolysis bullosa shows ongoing benefit
Earlier this year, the FDA declined to approve oleogel-S10 for use in EB, even after it extended its review by 3 months to include additional...
Latest News
Pandemic public health measures may have mitigated Kawasaki disease
Cohort study data suggest that actions to reduce the spread of COVID-19 also reduced exposure to Kawasaki disease triggers.
Guidelines
Myositis guidelines aim to standardize adult and pediatric care
These are the first guidelines on how to manage adult and pediatric patients with idiopathic inflammatory myopathies (IIM).
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.
From the Journals
IV gentamicin improves junctional epidermolysis bullosa in children
After treatment, each of the children had increased, sustained expression of laminin 332.
Conference Coverage
Dupilumab treats itch and clears lesions in prurigo nodularis patients
There are currently no Food and Drug Administration–approved systemic therapies for PN.
Conference Coverage
Novel isotretinoin ointment for congenital ichthyosis shows promise
“There is currently no FDA-approved drug for congenital ichthyosis,” Dr. Christopher G. Bunick said.