Rare Diseases
News from the FDA/CDC
FDA approves belimumab for children with lupus nephritis
Children with lupus-related kidney damage can now receive the B cell–reducing therapy that adults with lupus nephritis have been able to get for...
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.
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
Acute hepatitis cases in children show declining trend; adenovirus, COVID-19 remain key leads
Adenovirus remains the most commonly detected virus in acute hepatitis in children, found in 53% of cases overall.
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.
News
FDA approves setmelanotide for obesity in Bardet-Biedl syndrome
The FDA cleared setmelanotide (Imcivree) for people age 6 and older with obesity due to a rare genetic disorder.
Conference Coverage
Biomarkers may help to predict persistent oligoarticular JIA
Analysis of a small group of samples revealed that the selected biomarkers more accurately predicted persistent disease than disease likely to...
ID Consult
Monkeypox: What’s a pediatrician to do?
Given the media coverage of the outbreak, pediatricians should be prepared for questions from patients and their parents.
Guidelines
Updated pediatric uveitis recommendations advise on expanded treatment options
The recommendations represent an update of 2018 consensus-based recommendations from the SHARE Initiative and the 2019 American College of...
News
FDA expands indication for spinal muscular atrophy drug
After 12 months of risdiplam treatment, the majority of presymptomatic infants with SMA reached key motor milestones.
Feature
Meet a miracle: Man with trisomy 13 to celebrate 20th birthday
Lloyd Tyler Rochez was born in 2002 with a genetic disorder that can involve severe learning problems and health woes that affect nearly every...