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ARRA Investments in Pediatrics

Public Health Burden
Understanding child development at the earliest stages is essential to ensuring healthy babies. As every child grows, developmental processes continue, with lifelong impacts on learning, behavior, and health. Because of these developmental processes, children often may require different, specialized dosing and treatment even when they have the same disorders as adults. ARRA supported research is deepening scientists’ knowledge of human development, and then translating these insights into better prevention and treatment efforts specifically designed for children.

Preventing and Treating Birth Defects and Inherited Disorders
Understanding both normal and atypical development is crucial for determining the causes of birth defects  and, in turn, developing prevention and treatment strategies.  ARRA-funded grants are helping to advance our knowledge of the developmental mechanisms behind a variety of disorders.
  • Limb anomalies are some of the most common birth defects in humans. ARRA support is helping scientists understand the mechanisms underlying normal limb development, to identify implications for potential therapies.1
  • Researchers will study insect embryonic development to gain insight into specific changes in cell shape.  The results may help improve understanding of human neural tube formation and congenital neural tube defects.2
  • Scientists will investigate biological mechanisms that control the formation of sensory organs in an animal model.  This research may give researchers insights into birth defects related to sensory organs in humans.3
Science, Technology, Engineering and Mathematics (STEM) Education
Science, technology, engineering and mathematics education is fundamental to the health and well-being of the nation.  The ARRA program provides a unique opportunity to enrich the science base in mathematics and science cognition and learning, and to apply this knowledge to develop practical interventions for tomorrow’s classrooms.
  • Early-life fine motor skills may predict later-life math skills.   Researchers will evaluate the effects of two fine motor skill interventions on math achievement in first-graders, including disadvantaged children.4
  • To improve and guide preschool math education, researchers will examine how well geometry tests for 3-yr old children predict their overall math abilities 14-months later.5
  • Researchers will examine math problem solving skills and attitudes, following an established intervention, in a diverse group of third graders who have math difficulties.6
Specialized Treatment for Pediatric Populations
Rigorous clinical research is urgently needed to assess the safety and effectiveness of drugs currently used in children.  Nearly 80 percent of prescription drugs lack scientific data on appropriate dosing, efficacy, and safety for neonates, infants, children, or adolescents, although physicians do prescribe them "off label" for pediatric use. ARRA funding will support research on drugs currently used to treat children with conditions such as sickle cell anemia, asthma, spinal muscular atrophy, and pediatric hypertension, among others.
  • Investigators will use an animal model to study ways to enhance immune response, to improve the current therapy for newborns with Pompe disease, a serious neuromuscular disorder.7
  • Researchers will design and validate a fully implantable, magnetically levitated left ventricular assist device for infants and children.8
  • Inflammatory bowel disease (IBD), a chronic and unpredictable disorder, is increasing in children.  Researchers will assess behavioral factors in pediatric IBD, to improve IBD treatment.9
  • Scientists will develop and validate a tool to help physicians create personalized treatment plans, based on a child's genetic makeup and other factors, for the pediatric anticoagulant warfarin.10
  • Scientists will investigate cellular mechanisms of cyst formation and also a new drug therapy in animal models of the two most important, incurable genetic liver and kidney diseases in infants.11
  • Spinal muscular atrophy is a leading genetic cause of infant mortality and morbidity.  This study will use adult stem cells to develop nerve and muscle cells that may be helpful in treating SMA.12
  • To ultimately improve how physicians prescribe cough medicine for children at different developmental stages, the investigator will study a gene involved in metabolizing many drugs used in children.13
  • Using an animal model, researchers will investigate how brain injury happens after asphyxia or cardiac arrest in children, in order to help find ways to prevent brain injury in children after drowning.14

  1. 1R15HD060087-01 -- The role of Fgf and Wnt5a signaling in directed outgrowth of the limb mesenchyme -- Barrow, Jeffrey (UT)
  2. 1R15HD059957-01 -- The developmental function and evolutionary history of the Drosophila folded gast -- Hoang, Rachel (PA)
  3. 1R15HD060011-01 -- Ofactory development in moths: a novel system for studying pattern formation -- Kenyon, Kristy (NY)
  4. 1RC1HD063534-01 -- Improving Fine Motor Skill Development to Promote Mathematical Ability -- Grissmer, David (VA)
  5. 1RC1HD063497-01 -- Shape Up Preschoolers: Geometric Sense Predicts Future Mathematics Achievement -- Golinkoff, Roberta (DE)
  6. 1RC1HD063996-01 -- Improving Problem-Solving Performance of Students with Mathematics Difficulties -- Jittendra, Asha (MN)
  7. 1R01HD054795-01A2 -- Mechanisms for immune tolerance in Pompe Disease -- Koeberl, Dwight (NC)
  8. 1RC1HD064041-01 -- Development of the PediaFlow Pediatric Ventricular Assist Device -- Wearden, Peter (PA)
  9. 1R21HD058828-01A1 -- Development of the Pediatric IBD Behavioral Health Registry -- Lobato, Debra (RI)
  10. 1R01HD061312-01 -- Warfarin Pharmacogenetics in Pediatric Patients -- Hines, Ronald (WI)
  11. 1R03HD059878-01A1 -- Intracellular calcium in the treatment of polycystic kidney and liver diseases -- Gradilone, Sergio (MN)
  12. 1R21HD060899-01 -- Motor neuron generation from SMA patient-dervied induced pluriopotent stem cells -- Ebert, Allison (WI)
  13. 1R01HD058556-01 -- Exogenous and Endogenous Biomarkers of CYP2D6 Variability in Pediatrics -- Leeder, James (MO)
  14. 1R21HD058846-01A1 -- COX2-Derived Cyclopentenone Prostaglandins Exacerbate Hypoxic Ischemic Brain Injury -- Hickey, Robert (PA)

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