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Pediatric

All articles tagged with #pediatric

FDA Clears First Gene Therapy for Severe LAD-I in Children
health4 hours ago

FDA Clears First Gene Therapy for Severe LAD-I in Children

The FDA approved Kresladi (marnetegragene autotemcel), the first gene therapy for severe Leukocyte Adhesion Deficiency Type I in pediatric patients without an HLA-matched donor, using autologous gene-modified stem cells to restore ITGB2 function and CD18/CD11a on white blood cells. The treatment is given as a single IV infusion after conditioning, based on biomarker improvements in an open-label trial, with post-marketing confirmatory studies required and safety monitoring for cytopenias and infections.

Texas teen loses three limbs after flu-triggered pneumonia complications
health6 days ago

Texas teen loses three limbs after flu-triggered pneumonia complications

A 13-year-old Kerrville, Texas, girl named Kaydin Ruiz developed life-threatening flu complications when influenza led to a severe strep pneumonia infection that caused clotting and organ failure, resulting in the amputation of three limbs; she remains hospitalized at Texas Children’s Hospital as her family raises awareness of flu risks and doctors urge vaccination to prevent such severe outcomes.

New drug brings life-changing relief to children with Dravet syndrome
health22 days ago

New drug brings life-changing relief to children with Dravet syndrome

A new drug, zorevunersen, delivered by spinal infusion, is showing strong promise for Dravet syndrome in children by boosting brain sodium channel activity and dramatically reducing seizures; early trials report up to 90% fewer seizures, with patients like Freddie Truelove moving from hundreds of daily seizures to a few per week and regaining normal activities. UK and US trials involve young children, starting from age two, at several hospitals, and while results are encouraging, Phase 3 data are needed before it becomes widely available.

TikTok trend sparks dangerous mealtime dare: 9-year-old severely burned from microwaving NeeDoh toy
health1 month ago

TikTok trend sparks dangerous mealtime dare: 9-year-old severely burned from microwaving NeeDoh toy

A 9-year-old Illinois boy was severely burned after a TikTok trend led him to microwave a NeeDoh sensory toy. He suffered facial and hand burns and an eye swelling, was treated at Loyola Medicine’s Burn Center, and has since returned home. Loyola notes it’s the fourth NeeDoh-related burn this year, and the toy’s manufacturer warns not to heat or microwave the toy.

health2 months ago

ELEVIDYS Slows Duchenne Progression in Three-Year EMBARK Results

Sarepta reports three-year topline results from EMBARK showing ELEVIDYS significantly slows disease progression in ambulatory Duchenne patients treated at ages 4–7. By year 3, NSAA remained above baseline, with a 73% slowing of progression by Time to Rise and a 70% slowing by 10-meter walk/run versus a pre-specified external control; safety signals align with prior data, including a boxed warning for acute liver injury and related risks, with no new safety concerns observed.

Mother urges flu vaccination after daughter's death from flu complications
health2 months ago

Mother urges flu vaccination after daughter's death from flu complications

A 4-year-old girl died from flu complications after a co-infection with adenovirus led to pneumonia and sepsis. Her mother speaks out to urge other parents to vaccinate against the flu, noting CDC data showing pediatric flu deaths and that most of the affected children were not fully vaccinated, while doctors warn that flu can deteriorate quickly in children and emphasize vigilance for warning signs.

Multiple States Report First Pediatric Flu Deaths of the Season
health2 months ago

Multiple States Report First Pediatric Flu Deaths of the Season

Greene County, Ohio, reports its first pediatric flu death of the 2025-26 season, involving a healthy 16-year-old girl from Beavercreek. Health officials emphasize the importance of vaccination and early treatment, noting that while most flu cases are mild, severe symptoms require urgent medical attention. Vaccination is still recommended despite the circulating strain, with ample supply available.