A5061181330- Glioma Diagnosis and Treatment
- Cystic Fibrosis Research Advances
- Neuroblastoma Research and Treatments
- Hedgehog Signaling Pathway Studies
- Brain Metastases and Treatment
- TGF-β signaling in diseases
- Mycobacterium research and diagnosis
- Testicular diseases and treatments
- Immunotherapy and Immune Responses
- Glycosylation and Glycoproteins Research
- Tracheal and airway disorders
- Pediatric health and respiratory diseases
- CAR-T cell therapy research
- Child Nutrition and Feeding Issues
- Cancer-related Molecular Pathways
- Antimicrobial Resistance in Staphylococcus
- Inhalation and Respiratory Drug Delivery
- Vascular Malformations Diagnosis and Treatment
- Epilepsy research and treatment
- RNA regulation and disease
- Genetics and Neurodevelopmental Disorders
- Tuberculosis Research and Epidemiology
- Radiopharmaceutical Chemistry and Applications
- Hepatocellular Carcinoma Treatment and Prognosis
- Metabolism and Genetic Disorders
Vertex Pharmaceuticals (United States)
2022-2023
Johns Hopkins University
2014-2021
Mount Sinai Health System
2021
Johns Hopkins Medicine
2017-2020
Johns Hopkins Hospital
2020
Pulmonary and Critical Care Associates
2017
Vanderbilt University Medical Center
1990-2009
Good Samaritan Hospital
2007
Children's Hospital of Illinois
2006
St. Jude Children's Research Hospital
2005
Purpose To evaluate response rate, event-free survival (EFS), and toxicity of two chemotherapeutic regimens for treatment children younger than 36 months with malignant brain tumors to estimate control intervals without irradiation in no residual tumor after initial surgery induction chemotherapy delayed patients or metastatic disease at diagnosis. Patients Methods were randomly assigned one (vincristine, cisplatin, cyclophosphamide, etoposide v vincristine, carboplatin, ifosfamide,...
A 58-year-old woman was admitted with symptoms of coronavirus disease-2019. She subsequently developed mixed shock, and an echocardiogram showed mid-distal left ventricular hypokinesis apical ballooning, findings typical stress, or takotsubo, cardiomyopathy. Over the next few days her function improved, further supporting reversibility acute stress (Level Difficulty: Beginner.)
Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis at least one F508del-CFTR allele a phase 3, open-label, single-arm study. Objectives: To further evaluate the efficacy safety of ELX/TEZ/IVA age heterozygous for F508del minimal function CFTR mutation (F/MF genotypes) randomized, double-blind, placebo-controlled 3b trial. Methods: Children were randomized...
PURPOSE: To determine the optimal cutoff level of fluorine-18-labeled fluorodeoxyglucose (FDG) uptake in differentiation low-grade from high-grade cerebral tumors at position emission tomography (PET). MATERIALS AND METHODS: The authors retrospectively reviewed images PET, magnetic resonance imaging, and computed performed 58 consecutive patients with histologically proved brain tumors. There were 32 (20 gliomas) 26 (18 gliomas). RESULTS: best FDG ratios was 1.5 for tumor-to-white matter...
Carboplatin, a cisplatin analogue, was administered as an intravenous (IV) one-hour infusion in 4-consecutive weekly dose schedule to 44 patients with recurrent childhood brain tumors. Twenty-four were registered on our phase I, and 20 II studies. The maximum tolerable derived from I study 210 mg/m2/wk solid tumors, the recommended for subsequent pediatric studies 175 mg/m2/wk. This 14 all study. Nine of 36 (25%) evaluable combined experienced objective responses median duration 10+ months....
ADVERTISEMENT RETURN TO ISSUEPREVArticleNEXTCell-type-specific and site-specific N-glycosylation of type I II human tissue plasminogen activatorRaj B. Parekh, Raymond A. Dwek, Jerry R. Thomas, Ghislain Opdenakker, Thomas W. Rademacher, Arthur J. Wittwer, Susan C. Howard, Rickey Nelson, Ned Siegel, M Jennings, Nikos Harakas, Joseph FederCite this: Biochemistry 1989, 28, 19, 7644–7662Publication Date (Print):September 1989Publication History Published online1 May 2002Published inissue 19...
We studied three cases (two of them siblings) with ocular and cerebral dysgenesis (Walker-Warburg syndrome). The histologic changes suggest that the disorder results from a sclerosing meningoencephalitis active through second third trimesters, but different typically encountered known congenital pathogens. This illness was first in 1930s has been reported increasing frequency since 1970. risk recurrence among siblings exceeds 50%, predilection for involving consecutive siblings, high...
In July 2015, the U.S. Food and Drug Administration approved lumacaftor/ivacaftor for use in patients with cystic fibrosis (CF). This drug targets primary defect CFTR protein that is conferred by F508del mutation.As there limited experience this therapy outside of clinical trials, study aims to examine new a population CF.Retrospective cohort individuals followed at Johns Hopkins CF Center who initiated treatment lumacaftor/ivacaftor. Patients were from 1 year before initiation up 11 months...
Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) ≥ 1 F508del-CFTR allele Phase 3 clinical trials. ELX/TEZ/IVA treatment led improved lung function, increases percent predicted forced expiratory volume second (ppFEV1) Cystic Fibrosis Questionnaire-Revised respiratory domain score. Here, we evaluated the impact of on rate function decline over time by comparing changes ppFEV1 participants from trials a matched group CF US...
Abstract Low‐grade astrocytomas, anaplastic astrocytomas and glio‐blastomas in vitro were found to ubiquitously produce the mRNA of transforming growth factor‐β (TGFβ). TGFβ 1 2 expressed a lesser degree among hyperdiploid malignant gliomas. By radioreceptor assay conditioned medium, was secreted predominantly latent form, both active or only form within panel low‐grade The receptor (types I, II III) evident glioma lines. Many near‐diploid gliomas growth‐inhibited by . Most glioblastomas...
PURPOSE: This Children’s Cancer Group group-wide phase II trial evaluated the efficacy and toxicity of two chemotherapy arms administered before hyperfractionated external-beam radiotherapy (HFEBRT). PATIENTS AND METHODS: Thirty-two patients with newly diagnosed brainstem gliomas were randomly assigned to regimen A 31 B. Regimen comprised three courses carboplatin, etoposide, vincristine; B cisplatin, cyclophosphamide, vincristine. Both included granulocyte colony-stimulating factor....
Abstract With the exception of macro‐orchidism, three families with X‐linked mental retardation showed diagnostic concordance clinical features among affected males. Since macro‐orchidism was a variable feature otherwise identically males in one family, we question existence separate entity characterized only by testicular enlargement. The X chromosome marker Lubs expressed, under culture conditions Sutherland, lymphocytes two families, and other without megalotestes. Two members third...
This edition follows the first by 5 years and incorporates recent advances in basic sciences into practice of child neurology. In a period increasingly dominated strictly defined review article monograph, authors continue to work specific context whole pediatric neuroscience. The majority chapters are solely authored an acknowledged leader field, thus affording his or her organizational perspective overview. text remains divided four parts, which include clinical assessment (pages 3 through...
Six adults and 2 children with focal inhibitory motor seizures (ictal paralysis) were evaluated during a 4-year period. Paresthesias at seizure onset occurred some in all patients, clonic activity followed paralysis 4. EEG-CCTV recordings of the patients showed that ictal coincided an discharge starting one centroparietal area. MRI structural lesions six patients. One patient normal scan had right hypometabolism on PET. Inhibitory must be differentiated from transient ischemic attacks...