Open Access. Powered by Scholars. Published by Universities.®
- Keyword
-
- Adult; adverse outcome; child; chronic kidney disease; clinical assessment; clinical effectiveness; controlled clinical trial; controlled study; drug efficacy; drug safety; female; growth disorder; growth hormone deficiency; hormonal therapy; human; major clinical study; male; Noonan syndrome; observational study; outcome assessment; pharmacoepidemiology; Prader Willi syndrome; review; short stature; small for date infant; study design; Turner syndrome; unspecified side effect (1)
- Agammaglobulinemia (1)
- Age Factors (1)
- Child (1)
- Child, Preschool (1)
-
- Development of frataxin gene expression measures for the evaluation of experimental treatments in Friedreich's ataxia. (1)
- Female (1)
- Genetic Predisposition to Disease (1)
- Humans (1)
- Immunoglobulins (1)
- Immunophenotyping (1)
- Infant (1)
- Male (1)
- Predictive Value of Tests (1)
- Preschool (1)
- Reference Values (1)
- Sex Factors (1)
- Time Factors (1)
Articles 1 - 3 of 3
Full-Text Articles in Medicine and Health Sciences
Phenotypic Parameters Predict Time To Normalization In Infants With Hypogammaglobulinemia., Robert C. Van Winkle, Walter W. Hauck, Stephen J. Mcgeady
Phenotypic Parameters Predict Time To Normalization In Infants With Hypogammaglobulinemia., Robert C. Van Winkle, Walter W. Hauck, Stephen J. Mcgeady
Department of Pediatrics Faculty Papers
PURPOSE: Infants with recurrent infection may be found to have hypogammaglobulinemia without impaired specific antibody responses. Many will be diagnosed with transient hypogammaglobulinemia of infancy.
METHODS: This study used a parametric survival analysis of 100 infants with hypogammaglobulinemia to predict time to normalization.
RESULTS: Aggregate initial immunoglobulins (IgG + IgA + IgM), as a percentage of age-adjusted normal, predicted time to resolution: median time to resolution for the infants in the lowest quartile of aggregate levels (≤81 % of age-adjusted lower limits) was greater than 5 years, with 34 % resolving in 3 years. For infants in the highest quartile …
The Nordinet® International Outcome Study And Novonet® Answer Program®: Rationale, Design, And Methodology Of Two International Pharmacoepidemiological Registry-Based Studies Monitoring Long-Term Clinical And Safety Outcomes Of Growth Hormone Therapy (Norditropin®)., Charlotte Höybye, Lars Sävendahl, Henrik Thybo Christesen, Peter Lee, Birgitte Tønnes Pedersen, Michael Schlumpf, John Germak, Judith Ross
The Nordinet® International Outcome Study And Novonet® Answer Program®: Rationale, Design, And Methodology Of Two International Pharmacoepidemiological Registry-Based Studies Monitoring Long-Term Clinical And Safety Outcomes Of Growth Hormone Therapy (Norditropin®)., Charlotte Höybye, Lars Sävendahl, Henrik Thybo Christesen, Peter Lee, Birgitte Tønnes Pedersen, Michael Schlumpf, John Germak, Judith Ross
Department of Pediatrics Faculty Papers
OBJECTIVE: Randomized controlled trials have shown that growth hormone (GH) therapy has effects on growth, metabolism, and body composition. GH therapy is prescribed for children with growth failure and adults with GH deficiency. Carefully conducted observational study of GH treatment affords the opportunity to assess long-term treatment outcomes and the clinical factors and variables affecting those outcomes, in patients receiving GH therapy in routine clinical practice.
DESIGN: The NordiNet® International Outcome Study (IOS) and the American Norditropin® Studies: Web Enabled Research (ANSWER Program®) are two complementary, non-interventional, observational studies that adhere to current guidelines for pharmacoepidemiological data.
PATIENTS: The studies …
Development Of Frataxin Gene Expression Measures For The Evaluation Of Experimental Treatments In Friedreich's Ataxia., Heather L Plasterer, Eric C Deutsch, Matthew Belmonte, Elizabeth Egan, David R Lynch, James R Rusche
Development Of Frataxin Gene Expression Measures For The Evaluation Of Experimental Treatments In Friedreich's Ataxia., Heather L Plasterer, Eric C Deutsch, Matthew Belmonte, Elizabeth Egan, David R Lynch, James R Rusche
Department of Pediatrics Faculty Papers
BACKGROUND: Friedreich ataxia is a progressive neurodegenerative disorder caused by GAA triplet repeat expansions or point mutations in the FXN gene and, ultimately, a deficiency in the levels of functional frataxin protein. Heterozygous carriers of the expansion express approximately 50% of normal frataxin levels yet manifest no clinical symptoms, suggesting that therapeutic approaches that increase frataxin may be effective even if frataxin is raised only to carrier levels. Small molecule HDAC inhibitor compounds increase frataxin mRNA and protein levels, and have beneficial effects in animal models of FRDA.
METHODOLOGY/PRINCIPAL FINDINGS: To gather data supporting the use of frataxin as a …