A5000102952- Lysosomal Storage Disorders Research
- Glycogen Storage Diseases and Myoclonus
- Trypanosoma species research and implications
- Neurogenetic and Muscular Disorders Research
- Fetal and Pediatric Neurological Disorders
- Family and Disability Support Research
- Heart Failure Treatment and Management
- Autism Spectrum Disorder Research
- Cardiac Arrest and Resuscitation
- Emergency and Acute Care Studies
- Metabolism and Genetic Disorders
- Thermal Regulation in Medicine
- Neonatal and fetal brain pathology
- Opioid Use Disorder Treatment
- RNA regulation and disease
- Hereditary Neurological Disorders
- Advanced Neuroimaging Techniques and Applications
- Williams Syndrome Research
- Thermoregulation and physiological responses
- Cardiovascular Function and Risk Factors
- Biomedical Research and Pathophysiology
- Neurological diseases and metabolism
- Cystic Fibrosis Research Advances
- Genomics and Rare Diseases
- Acute Lymphoblastic Leukemia research
University of Minnesota Medical Center
2019-2025
University of Minnesota
2009-2022
BioMarin (United States)
2017-2022
Cornell University
2021
Emory University
2019
UCSF Benioff Children's Hospital
2019
Birmingham Children's Hospital
2019
Hospital for Sick Children
2019
John Peter Smith Hospital
2014-2016
Health Net
2014-2016
The LACE index has been used to predict the risk of unplanned readmission within 30 days after hospital discharge in both medical and surgical patients. aim this study is validate accuracy using CHF This was a retrospective study. score calculated on each patient who admitted due an acute exacerbation. Operational clinical variables were collected from patients including basic characteristics, length hospitalization, comorbidities, number previous ED visits past 6 months before admission,...
ObjectivesTo characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identify potential endpoints for future treatment trials.Study designChildren with a confirmed diagnosis MPS IIIA, functioning above developmental age 1 year, were followed up to 2 years. Cognitive status brain atrophy assessed by standardized tests volumetric magnetic resonance imaging, respectively. Liver spleen volumes cerebrospinal fluid urine biomarker levels measured.ResultsTwenty-five...
To evaluate empirical therapy with trimethoprim-sulfamethoxazole or doxycycline for outpatient skin and soft tissue infections in an area of high prevalence methicillin-resistant Staphylococcus aureus, a randomized, prospective, open-label investigation was performed. The overall clinical failure rate 9%, all failures occurring the group. However, there no significant difference between that therapy.
Abstract Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disorder leading to deleterious brain effects. While animal models suggested that MPS severely affects white matter (WM), whole‐brain diffusion tensor imaging (DTI) analysis was not performed due MPS‐related morphological abnormalities. 3T DTI data from 28 severe IH, treated with hematopoietic stem cell transplantation—HSCT), 16 attenuated patients IA) enrolled under the study protocol NCT01870375 , and 27...
Objective: To determine the effects of body temperature, ethanol use, electrolyte status, and acid‐base status on electrocardiograms (ECGs) hypothermic patients. Methods: Prospective, two‐year, observational study patients presenting to an urban ED with temperature ≤95°F (≤35°C). All had at least one ECG obtained. Electrocardiograms were interpreted by a cardiologist blinded patient's temperature. J‐point elevations known as Osborn waves defined present if they 1 mm in height two consecutive...
(1) Develop a methodology for obtaining reliable cognitive and developmental data in children with neurodegenerative disease impairment turn monitor state treatment outcomes. (2) Demonstrate validity of age-equivalent scores.We present guidelines accurate test scores low-functioning behaviorally disruptive pediatric patients, followed by method validation study: using disease-specific protocols to assess salient aspects the known phenotype, selecting appropriate tests, (3) managing behavior,...
Our goal was to describe the neurobehavioral phenotype in mucopolysaccharidosis Type IIIB (MPS IIIB). Parents report that behavioral abnormalities are a major problem MPS III posing serious challenges parenting and quality-of-life for both patient parent. previous research on IIIA identified autistic symptoms, Klüver-Bucy-type syndrome as indicated by reduced startle loss of fear associated with amygdala atrophy. We hypothesized would manifest similar attributes when assessed same...
Advances in medical treatment have prolonged the lives of children with Hurler syndrome or mucopolysaccharidosis I requiring increased attention to assessment their long-term outcomes and functional abilities. Adaptive functions are critical for understanding after developing focused interventions. We investigated development various adaptive who had hematopoietic stem cell transplant (HSCT) risk factors that associated these functions. examined 41 3 more Vineland Behavior Scales records...
The behavioral, adaptive and quality of life characteristics attenuated mucopolysaccharidosis type II (MPS II) have not been well studied. Understanding changes over time in the phenotype may assist helping achieve better outcomes long-term function. This longitudinal study investigates these relation to age, somatic disease burden, IQ. Specifically, burden is a major challenge for patients, even with treatment enzyme replacement therapy.
Mucopolysaccharidosis type III is a progressive, neurodegenerative lysosomal storage disorder for which there currently no effective therapy. Though numerous potential therapies are in development, several challenges to conducting clinical research this area. We seek make recommendations on the approach MPS III, including selection of outcome measures and trial endpoints, order improve quality impact An international workshop involving academic researchers, experts industry groups was held...