Medical Specialties Commons^™

Global Cns Gene Transfer For A Childhood Neurogenetic Enzyme Deficiency: Canavan Disease., Paola Leone, Christopher G Janson, Scott J Mcphee, Matthew J During

Department of Neurosurgery Faculty Papers

The neurogenetic prototypic disease on which we chose to test our gene therapy strategy is Canavan disease (CD). CD is an autosomal recessive leukodystrophy associated with spongiform degeneration of the brain. At present the disease is uniformly fatal in affected probands. CD is characterized by mutations in the aspartoacylase (ASPA) gene, resulting in loss of enzyme activity. In this review, recent evidence is summarized on the etiology and possible treatments for CD. In particular, we discuss two gene delivery systems representing recent advances in both viral and liposome technology: a novel cationic liposome-polymer-DNA (LPD) complex, DCChol/DOPE-protamine, as well as recombinant …

Nebulized Nitroglycerin In Children With Pulmonary Hypertension Secondary To Congenital Heart Disease, Hatim Omar, Fangqi Gong, Mei Y. Sun, Stanley Einzig

Pediatrics Faculty Publications

Pulmonary hypertension continues to be a major cause of morbidity and mortality, despite new treatments. Since inhaled nitric oxide has been reported to be effective in some cases, we investigated using nebulized nitroglycerine to treat pulmonary hypertension in children with congenital heart disease. Four children (ages 6-72 months) with severe pulmonary hypertension secondary to congenital heart disease (all with membranous ventricular septal defect, undergoing cardiac catheterization) were given 3 cc of nebulized normal saline over 10 min as placebo control, followed by nebulized nitroglycerine (20 micrograms/Kg in 3 cc normal saline). Normal saline administration did not elicit any change, but …

Home Observation For Asymptomatic Coin Ingestion: Acceptance And Outcomes. The New York State Poison Control Center Coin Ingestion Study Group., Gregory P. Conners, D J. Cobaugh, R Feinberg, R Lucanie, T Caraccio, C M. Stork

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVES: To obtain preliminary estimates of the acceptance rate and the frequency of adverse outcomes, and to identify issues related to acceptance, associated with management of asymptomatic pediatric coin ingestion by home observation, in preparation for a large-scale prospective study.

METHODS: Scripted telephone follow-up of callers who had reported asymptomatic pediatric coin ingestions to one of five poison control centers six to 36 months previously, which had been managed by home observation.

RESULTS: Of the 67 callers enrolled, 41 (67%) reported contacting a physician regarding the coin ingestion, despite home observation instruction by poison control center personnel. Those who did …