Rare Diseases
Latest News
PIK3CA-Related Overgrowth Spectrum (PROS): 5 Things to Know
Comprehensive and regular monitoring is essential to address the diverse and evolving clinical manifestations of PROS.
Latest News
When Should a Pediatrician Suspect a Rare Disease?
“Patients with rare diseases have common symptoms. What needs to be done is to prioritize those symptoms that behave abnormally, that have an...
News from the FDA/CDC
Second FDA-Approved Tocilizumab Biosimilar Has Intravenous, Subcutaneous Formulations
It’s unclear when the new tocilizumab products will be commercially available.
Latest News
Oral Transmission of Chagas Disease Has Severe Effects
"A myth I would like to dispel is that Chagas disease is not being transmitted in the United States."
Latest News
National Rapid Genome Testing Program Benefits NICU Care
Rapid trio genome sequencing diagnosed 50% of the neonates with disease-causing genetic variants.
News from the FDA/CDC
FDA Approves 10th Humira Biosimilar, With Interchangeability
Adalimumab-ryvk (Simlandi) is the first adalimumab biosimilar in a high-concentration formulation to receive interchangeability status from the...
Latest News
Lichen Sclerosus: The Silent Genital Health Concern Often Missed
Lichen sclerosus, which causes pain, itching, and skin changes, most often effects the skin of the genital and anal regions.
Latest News
Study: Lifetime Cost of Vyjuvek Gene Therapy for DEB Could Be $15-$22 Million
"The wider indication granted by the FDA may lead to friction between payers on the one hand and patients and physicians on the other," the...
Latest News
Hair Loss in Children: How to Spot and Treat Different Causes
Most children with alopecia areata are healthy and do not need extensive screening laboratory testing. However, one exception is if thyroid...
Latest News
FDA Approves Topical Gel For Wounds Associated With JEB and DEB
The FDA has approved a topical gel containing birch triterpenes for the treatment of partial thickness wounds in patients 6 months and older with...
Conference Coverage
Teen and young adult rheumatology patients report gaps in sexual health counseling
In a new survey, only half of teens and young adults on teratogens said they had been counseled on the reproductive risk for their medications.