Rare Diseases
From the Journals
Consensus statement issued on retinoids for ichthyosis, disorders of cornification
To date, no specific guidance has addressed the safety, efficacy, or overall precautions for their use in the pediatric population.
From the Journals
Data call for biologics trials in undertreated juvenile arthritis subtype
Many children with enthesitis-related arthritis have significant disease burden despite treatment with biologics currently approved for adults...
From the Journals
Analysis characterizes common wound microbes in epidermolysis bullosa
Methicillin resistance and mupirocin resistance was reported in 47% and 40% of patients tested.
News
FDA clears first drug for rare genetic causes of severe obesity
Setmelanotide (Imcivree) is indicated for weight management in adults and children as young as 6 years with severe obesity driven by genetic...
Opinion
A 4-year-old presented to our pediatric dermatology clinic for evaluation of asymptomatic "brown spots."
A 4-year-old presents with asymptomatic "brown spots."
Conference Coverage
Higher cardiovascular risks in Kawasaki disease persist 10-plus years
Researchers evaluated the incidence of myocardial infarction, stroke, death, and more in a follow-up study of Kawasaki disease survivors, compared...
Conference Coverage
Gene-replacement therapy shows promise in X-linked myotubular myopathy
An investigational gene therapy decreases ventilator dependence and may improve motor development in children with a rare, life-threatening...
Conference Coverage
Birch bark derivative gel found effective for EB, in phase 3 study
"This is the first large clinical trial with placebo of a topical treatment that's worked for this terrible disease."
Conference Coverage
Ataluren delays disease milestones in patients with nonsense mutation DMD
Patients who received ataluren in addition to standard of care were 2.2 years older when they lost ambulation, compared with patients receiving...
From the Journals
JIA arthritis and uveitis flares ‘often run parallel’
Worsening arthritis in children with juvenile idiopathic arthritis could be a sign for needing a prompt eye examination.
Conference Coverage
Nusinersen provides continued benefits to presymptomatic children with SMA
Most children developed normally in an interim analysis of the NURTURE trial that did not find any new safety concerns.