A5014211264- Cystic Fibrosis Research Advances
- Adolescent and Pediatric Healthcare
- Neonatal Respiratory Health Research
- Tracheal and airway disorders
- Child Nutrition and Feeding Issues
- Childhood Cancer Survivors' Quality of Life
- Vascular Anomalies and Treatments
- Diabetes Management and Research
- Global Maternal and Child Health
- Dysphagia Assessment and Management
- Family and Disability Support Research
- Neurotransmitter Receptor Influence on Behavior
- Nematode management and characterization studies
- Vestibular and auditory disorders
- Lymphoma Diagnosis and Treatment
- Salivary Gland Disorders and Functions
- Asthma and respiratory diseases
- Advanced Biosensing Techniques and Applications
- Emergency and Acute Care Studies
- Child and Adolescent Health
- Inhalation and Respiratory Drug Delivery
- Pulmonary Hypertension Research and Treatments
- Ear Surgery and Otitis Media
- Cutaneous lymphoproliferative disorders research
- Breastfeeding Practices and Influences
Harvard University
2016-2025
Brigham and Women's Hospital
2015-2025
Boston Children's Hospital
2016-2025
Boston Children's Museum
2010-2025
Building Bridges
2024
Massachusetts General Hospital
2023
State Key Laboratory of Respiratory Disease
2023
Pulmonary Associates
2023
Imperial College London
2019
Boston University
2019
A new approach in the treatment of cystic fibrosis involves improving function mutant transmembrane conductance regulator (CFTR). VX-770, a CFTR potentiator, has been shown to increase activity wild-type and defective cell-surface vitro.We randomly assigned 39 adults with at least one G551D-CFTR allele receive oral VX-770 every 12 hours dose 25, 75, or 150 mg placebo for 14 days (in part 1 study) 250 28 2 study).At day 28, group subjects who received median change nasal potential difference...
The next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659–tezacaftor–ivacaftor), was developed to restore the function of Phe508del CFTR protein patients fibrosis.
A defect in bicarbonate secretion contributes to the pathophysiology of gastrointestinal complications patients with cystic fibrosis (CF). We measured pH, clinical outcomes, and intestinal transit profiles G551D mutation before after treatment ivacaftor, a CF transmembrane regulator channel (CFTR) potentiator.Observational studies ivacaftor effectiveness were conducted United States Canada. subset subjects ingested wireless motility capsule (n=10) that measures vivo both therapy 1 month...
Cystic fibrosis transmembrane conductance regulator modulators have revolutionized cystic (CF) care in the past decade. This study explores CF-related mortality trends US from 1999 to 2020. We extracted data CDC WONDER database. CF age-standardized rates (ASMRs) were identified by ICD-10 code E84 and stratified demographic geographical variables. Temporal analyzed using Joinpoint modeling. ASMRs decreased 1.9 1.04 per million population (p = 0.013), with a greater reduction recent years....
During the last decade, saliva has emerged as a potentially ideal diagnostic biofluid for noninvasive testing. In this paper, we present an automated, integrated platform useable by minimally trained personnel in field diagnosis of respiratory diseases using human sample specimen. platform, is loaded onto disposable microfluidic chip containing all necessary reagents and components required analysis. The then inserted into automated analyzer, SDReader, where multiple potential protein...
Abstract Background A large‐scale epidemiological study of 6088 individuals with cystic fibrosis (CF) and 4102 caregivers in nine countries documented elevated symptoms depression anxiety, leading to international guidelines for annual screening follow‐up. To facilitate national implementation, 84 CF programs funded a mental health coordinators (MHC). Implementation was evaluated after 1 year using the consolidated framework implementation research (CFIR) identify facilitators barriers....
Rationale: People with cystic fibrosis (CF) experience acute worsening of respiratory symptoms and lung function known as pulmonary exacerbations. Treatment intravenous antimicrobials is common; however, there scant evidence to support a standard treatment duration. Objectives: To test differing durations for CF Methods: STOP2 (Standardized Pulmonary Exacerbations 2) was multicenter, randomized, controlled clinical trial in exacerbations among adults CF. After 7-10 days treatment,...
The connection between agonist-induced desensitization and down-regulation of 5-hydroxytryptamine2A (5-HT2A) receptors was examined in a clonal cell line that stably expresses the 5-HT2A receptor. Brief (2-hr) prolonged (24-hr) exposure to agonist quipazine or 4-iodo-(2,5-dimethoxy)- phenylisopropylamine (DOI) diminished receptor-mediated phosphoinositide hydrolysis; no change receptor number affinity measured after 24 hr DOI quipazine. Immunohistochemical studies demonstrated 24-hr did not...
In this paper we show that a highly conserved aromatic residue, phenylalanine at the 340-position, is essential for ergoline binding to 5-hydroxytryptamine2A receptors. We hypothesized F340 was specific aromatic-aromatic interaction (e.g., pi-pi or hydrophobic) between phenyl moiety of and ring nucleus. To test hypothesis, eight point mutations adjacent (F340 F339) nonadjacent (F125) phenylanaines were made, using conservative (phenylalanine tyrosine) nonconservative leucine, alanine,...
Herbaspirillum species are not known to be human pathogens. We report on the identification of from cultures 28 persons with cystic fibrosis (CF). Most isolates were initially identified as members Burkholderia cepacia complex. Although role that these play in lung disease CF is known, their differentiation other important and has serious implications for clinical care patient well-being.
Context: Young adults with cystic fibrosis (CF) are at risk for low bone density and fractures, but the underlying alterations in microarchitecture that may contribute to their increased fracture currently unknown. Objective: The main goal of this study was use high-resolution peripheral quantitative computed tomography (HR-pQCT) characterize microarchitecture, volumetric mineral (vBMD), estimated strength radius tibia young CF compared healthy volunteers. Design Setting: This a...
Abstract Context The clinical utility and implications of continuous glucose monitoring (CGM) in cystic fibrosis (CF) are unclear. Objective We examined the correlation between CGM measures outcomes adults with CF, investigated relationship hemoglobin A1c (HbA1c) CGM-derived average (AG), explored that distinguish fibrosis–related diabetes (CFRD) from normal abnormal tolerance. Methods This prospective observational study included 77 CF who had HbA1c measured at 2 to 3 time points months...
Vocal biomarker-based machine learning approaches have shown promising results in the detection of various health conditions, including respiratory diseases, such as asthma.This study aimed to determine whether a respiratory-responsive vocal biomarker (RRVB) model platform initially trained on an asthma and healthy volunteer (HV) data set can differentiate patients with active COVID-19 infection from asymptomatic HVs by assessing its sensitivity, specificity, odds ratio (OR).A logistic...
In this study, the relationship between high-affinity agonist binding and second messenger production was examined at native mutant 5-hydroxytryptamine2A receptors. At receptors all agonists, with exception of quipazine, discriminated high- low-affinity states receptor, as determined by analysis competition assays. There no correlation ability selected agonists to label state augment phosphoinositide hydrolysis. Quipazine, which did not discriminate affinity behaved a full agonist. Similar...