CHILDREN'S HOSPITAL OF PHILADELPHIA

Antibiotic prophylaxis may protect kidneys of kids with vesicoureteral reflux during febrile UTIs, as per the RIVUR trial analysis. Children not on prophylaxis showed a significant eGFR decline, while those on prophylaxis did not. This suggests UTIs affect kidney function at a microscopic level, not just macroscopically monitored by DMSA scans.

Mitchell J. Weiss, MD, PhD, chair of St. Jude Children’s Research Hospital Department of Hematology, elected to National Academy of Medicine (NAM) for research in blood cell formation and treatment development for sickle cell disease and beta-thalassemia. Weiss has published over 170 research papers and joined six other St. Jude faculty members in NAM.

A $14M NIH grant funds Penn and CHOP research on CRISPR-based gene-editing therapies for urea cycle disorders, aiming to develop personalized treatments using prime editing technology, which could offer lifelong cures for rare metabolic diseases.

Pfizer pulled voxelotor, a sickle cell disease treatment, due to safety concerns, leaving patients and providers scrambling for alternatives. The community is rallying to maintain focus on finding a cure and increasing awareness of the disease.

The FDA approved self-administered nasal spray flu vaccine FluMist for adults and caregivers, available for ages 2-49. It contains live-attenuated virus, effective at 45%, similar to flu shots. Home use expected by 2025, currently administered by health providers. Not recommended for pregnant women, immune-compromised, or those on aspirin. Side effects include nasal congestion and fever. Easy self-administration aims to boost vaccination rates.

Virginia Tech researchers found that hypoxic conditions increase _Fusobacterium nucleatum_ infection in colorectal cancer cells, altering cancer gene expression, which could inform future drug development.

Biogen's nusinersen (Spinraza) showed efficacy at a higher dose than the FDA-approved regimen in the DEVOTE trial, with significant improvements on the CHOP-INTEND scale compared to a sham treatment. The higher dose was generally well-tolerated, though more serious adverse events were observed. Biogen plans to share detailed results with the SMA community and health authorities.

FDA approves neffy (epinephrine nasal spray) 2 mg for Type I Allergic Reactions, including anaphylaxis, in adults and children ≥30 kg, marking the first needle-free epinephrine treatment innovation in over 35 years.