Personalizing Enzyme Therapy to Improve Pancreatitis in Kids
Ochsner Health
When on pancreatic enzyme replacement therapy, the mean acute pancreatitis annual incidence rate decreased from 3.14 to 0.71
Children
Gene Therapy Improves Rare Neurodegenerative Disease in Trial
National Institutes of Health
Treatment for giant axonal neuropathy was well tolerated and slowed loss of motor function
Data-Driven Approach May Replace Rare Disease Clinical Trials
The Hospital for Sick Children
Rare diseases may be difficult to study and monitor for long periods due to small sample size and low occurrence
Noninvasive Biopsies Help Detect and Monitor Neuroblastoma
Princess Máxima Center for Pediatric Oncology
The simple blood tests for childhood cancers are being tested in the clinical setting for the first time
Next Generation Sequencing Will Advance Newborn Screening
Madhuri Hegde, PhD, FACMG
Genome sequencing uncovered risks of several pediatric-onset conditions that may benefit from early intervention
Combination Therapy Improves Rare Brain Tumors in Children
Great Ormond Street Hospital
Trials showed the effect of combination therapy on survival and quality of life in children with low- and high-grade gliomas
Measles Remains a Global Threat as Vaccination Coverage Declines
Centers for Disease Control and Prevention
Millions of children missed vaccinations during the COVID-19 pandemic, driving up the global number of cases
Trials Reveal First-Ever MDR-TB Treatment Safe for Kids and Adults
Desmond Tutu TB Centre at Stellenbosch University
Bayesian analysis of data from both trials showed that levofloxacin reduced the risk of developing TB by 60 percent
New Blood-Based Tool Rapidly Tests for TB in Children
Ludwig Maximilian University of Munich
An international research consortium has tested a rapid new analytical tool that needs just a blood sample from a fingerprick
FDA Approves Steroid-Like Drug for Duchenne Muscular Dystrophy
Children's National Hospital
Vamorolone offers a new treatment option with fewer side effects