All articles tagged with #pediatric
After a failed stem-cell transplant, a 13-month-old boy received a novel gene-therapy treatment and initially made progress, but a routine scan later revealed a golf-ball-sized brain tumor; scientists say the tumor may be linked to the gene-therapy viruses, highlighting the need to weigh life-saving benefits against rare but serious risks in such approaches.
An ICU doctor at Cincinnati Children’s Hospital details the rare hantavirus case of a 14-year-old patient who deteriorated rapidly, requiring intubation and five days on ECMO, a life-saving but high-risk heart–lung bypass. With no antiviral cure, treatment is supportive, and diagnosis is challenging because hantavirus is rare in Ohio and testing isn’t always rapid. Evie recovered after a month in hospital thanks to access to an ECMO-capable center and a skilled team, illustrating why quick transfer to such facilities is crucial and highlighting the ongoing need for research into therapies for this deadly disease.
Colorado has reported its eighth pediatric influenza death this season, the most in more than two decades aside from the 2009 H1N1 pandemic; five of the eight deaths were unvaccinated, with others lacking vaccination records. Officials say the season is severe and emphasize that flu vaccines offer protection, noting pediatric vaccination rates are similar to last year though rural areas lag. The flu can cause severe illness requiring ICU care, and nationwide pediatric flu deaths total 149 this season; vaccine uptake remains a challenge in younger adults and in several rural counties.
Rocket Pharmaceuticals announced that the FDA granted accelerated approval for KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell–based gene therapy for pediatric patients with severe LAD-I due to ITGB2 variants who lack an HLA-matched donor. Approval is based on increased neutrophil CD18/CD11a expression and will rely on longer-term follow-up data and a post‑marketing registry to confirm benefit. The FDA also awarded Rocket a Rare Pediatric Disease Priority Review Voucher, which the company plans to monetize. LAD-I is ultra-rare and causes recurrent life-threatening infections; safety concerns include infections during conditioning, veno-occlusive disease, engraftment failure, potential insertional oncogenesis, hypersensitivity, anti-retroviral interactions, and possible HIV test false positives, necessitating long-term monitoring. A conference call is planned for March 27, 2026.
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.
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.
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.
A two-year-old in Naples remains on life support in a stable but critical condition after a donor heart reportedly arrived frostbitten due to improper transport and direct contact with dry ice; prosecutors opened an investigation and doctors will decide if another transplant is viable as the family seeks a new donor heart.
The Wyoming Department of Health announced the death of a Fremont County child from influenza, part of a season dominated by the H3N2 strain; officials say flu can be serious, encourage annual vaccination, and outline preventive measures and antiviral treatment, noting nationwide pediatric deaths and that most affected children were not up to date on vaccines.
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.
A Snohomish County school-age teen died after contracting influenza, marking the state’s first pediatric flu death this season, with health officials reporting rising flu activity across western Washington and urging vaccination and preventive measures.
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.
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.
Colorado health officials confirm the second pediatric flu death of the 2025-2026 season, a high school-aged child from outside the metro Denver area who died in December; the state remains in the CDC’s 'very high' flu activity category, with 406 flu-related hospitalizations in the week ending Jan. 10 and 3,687 total since Oct. 1.
Virginia reports its first pediatric death from influenza this season (a child under 5 in the eastern region); health officials urge vaccination and continued precautions as flu activity fluctuates and vaccination rates remain below 30% among eligible Virginians.