Search results for "replacement therapy"
showing 10 items of 289 documents
Clinical outcomes of Hurler syndrome treated exclusively with enzyme replacement therapy from a young age
2015
Mental health perspectives of Hunter syndrome: Case reports of two biological siblings
2016
Hunter syndrome is a rare X-linked recessive disorder caused by deficiency of the lysosomal enzyme iduronate-2-sulphatase, leading to progressive accumulation of a substance called glycosaminoglycans in nearly all cell types, tissues, and organs. Hunter syndrome presents with facial dysmorphism, airway diseases, skeletal defects, cardiomyopathies, and neuropsychiatric manifestations. Mental subnormality is a cardinal feature in Hunter syndrome. This is a progressive cognitive decline that is not amenable to enzyme replacement therapy. Due to progressive cognitive decline, training the children to improve the adaptive functioning is a challenge that creates immense stress for the caregivers.…
Effect of hormone replacement therapy (HRT) on periodontal status of postmenopausal women.
2011
Summary Background The risks/benefits balance of hormone replacement therapy (HRT) is controversial. The aim of this study was to assess the periodontal status of a postmenopausal women group receiving HRT and to determine the effects of HRT on clinical measures of periodontal disease. Material/Methods Ninety-one postmenopausal women, 52 taking HRT (HRT+) and 39 not taking HRT (HRT−), completed the study. Clinical parameters measured included visible supragingival plaque, probing pocket depth (PD) and clinical attachment level (CAL). Gingival status was recorded as gingival bleeding on probing (BOP). Previous oral contraceptive use and current and past smoking status were also assessed. Res…
<p>Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis</p>
2019
Purpose Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9-67.3) for females (n=62), 34.4 (18.0-66.8) for males (n=90). With eGFR ≥60 mL…
mRNA-based therapeutics — developing a new class of drugs
2014
In vitro transcribed (IVT) mRNA has recently come into focus as a potential new drug class to deliver genetic information. Such synthetic mRNA can be engineered to transiently express proteins by structurally resembling natural mRNA. Advances in addressing the inherent challenges of this drug class, particularly related to controlling the translational efficacy and immunogenicity of the IVTmRNA, provide the basis for a broad range of potential applications. mRNA-based cancer immunotherapies and infectious disease vaccines have entered clinical development. Meanwhile, emerging novel approaches include in vivo delivery of IVT mRNA to replace or supplement proteins, IVT mRNA-based generation o…
Enzyme replacement therapy with agalsidase alfa in pregnant women with Fabry disease
2009
Role of menopause and hormone replacement therapy in sleep-disordered breathing
2020
There are suggestions that the loss of female sex hormones following menopause is critical for the development or progression of sleep-disordered breathing (SDB). We conducted a review of the literature on the role of menopause and hormone replacement therapy (HRT) in SDB risk. There is an increase in SDB during the menopausal transition period, but data on an effect beyond that of increasing age and changes in body habitus are weak or absent. Early community-based, observational studies reported a protective effect by HRT on SDB prevalence, but this could possibly be explained as a healthy user effect. Interventional studies of the effect of HRT on SDB are sparse, with only a few randomize…
Timing of Renal Replacement Therapy for Severe Acute Kidney Injury in Critically Ill Patients
2019
Acute kidney injury (AKI) affects many ICU patients and is responsible for increased morbidity and mortality. Although lifesaving in many situations, renal replacement therapy (RRT) may be associated with complications, and the appropriate timing of its initiation is still the subject of intense debate. An early initiation strategy can prevent some metabolic complications, whereas a delayed one may allow for renal function recovery in some patients without need for this costly and potentially dangerous technique. For years, most of the knowledge on this issue stemmed from observational studies or small randomized controlled trials. Recent randomized controlled trials have indicated that a w…