A surprise mimicker of a rare disorder: 13-year-old female with altered mental status ...

Jay T. Rubinstein, MD, PhD, emphasized that children with hearing loss should receive genetic testing, as the investigational DB-OTO gene therapy demonstrated positive results. Regeneron ...

On March 31, 2025, Ascendis Pharma A/S announced it has submitted a new drug application (NDA) to the FDA for navepegritide (TransCon CNP) to treat children with achondroplasia. 1 In a press release ...

Mesoblast's remestemcel-L (Ryoncil) was approved by the FDA on December 18, 2024 to treat SR-aGVHD in patients 2 months and older. Now, it is available for purchase. The FDA-approved allogeneic (donor ...

A look ahead to 5 key regulatory decisions scheduled to take place during the second quarter of 2025, from a monoclonal antibody to topical psoriasis treatments. As we transition into the second ...

Fitusiran is administered subcutaneously starting once every 2 months for patients aged 12 years and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors. The FDA has ...

Our editorial advisory board member Donna Hallas, , PPCNP-BC, CPNP, PMHS, FAANP, FAAN, highlights an article published in the March issue of Contemporary Pediatrics. Many parents may be using at-home ...

Several data points for lebrikizumab (EBGLYSS; Lilly), a monoclonal antibody targeting interleukin-13, were reported at the 2025 American Academy of Dermatology (AAD) meeting held in Orlando, Florida ...

A new clinical trial has found that omalizumab (Xolair; Genetech, Novartis) is more effective than oral immunotherapy (OIT) in treating multi-food allergy in individuals with severe allergic reactions ...

A 13-year-old female with a history of iron deficiency anemia presented with altered mental status preceded by 5 days of headache, vomiting, nausea, and worsening somnolence that was not responsive to ...

Recent research detailed that a child was most likely to be killed in a mass shooting by a parent or family member vs a stranger or peer. Study investigator Stephanie Chao, MD, breaks down these data.

In the phase 1/2 INSPIRE DUCHENNE trial, data from the first 3 patients dosed revealed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple ...