PubMed Journals Articles About "Sarepta Therapeutics Playing Nice With Over Rejection Duchenne" RSS

14:12 EST 5th December 2019 | BioPortfolio

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Showing "Sarepta Therapeutics playing nice with over rejection Duchenne" PubMed Articles 1–25 of 2,400+

Antecedents, development, adoption, and application of Duchenne's trocar for histopathologic studies of neuromuscular disorders in the nineteenth century.

Duchenne de Boulogne was one of the founders of clinical neurology. His name has been eponymically linked to the most common form of muscular dystrophy, originally described by him as pseudo-hypertrophic muscular paralysis or myo-sclerotic paralysis. Obtaining muscle biopsy specimens was essential to gain insight about the etiopathogenensis of the disease. Duchenne invented a novel instrument: , also known as "Duchenne's trocar," to perform muscle biopsies. Following Duchenne's design and instructions, a Pa...

NICE and Fair? Health Technology Assessment Policy Under the UK's National Institute for Health and Care Excellence, 1999-2018.

The UK's National Institute for Health and Care Excellence (NICE) is responsible for conducting health technology assessment (HTA) on behalf of the National Health Service (NHS). In seeking to justify its recommendations to the NHS about which technologies to fund, NICE claims to adopt two complementary ethical frameworks, one procedural-accountability for reasonableness (AfR)-and one substantive-an 'ethics of opportunity costs' (EOC) that rests primarily on the notion of allocative efficiency. This study i...

Implementation of NICE technology guidance on venous leg ulcers and diabetic foot ulcers: a conference report.

Implementation of evidence-based care is central to achieving good results. In the UK, this involves implementing guidance from the National Institute for Health and Care Excellence (NICE). Here, , consultant editor, reports on a conference that outlined recently published NICE guidance on UrgoStart for treating leg ulcers and diabetic foot ulcers, and described how to incorporate this into everyday practice to drive significant cost savings and improve patient outcomes.

Orofacial muscles may be affected in early stages of Becker muscular dystrophy: a preliminary study.

Dysphagia is reported in patients with Duchenne or Becker muscular dystrophy. Our clinical experience suggests that, compared to Duchenne patients, impaired mastication and swallowing occur early in Becker patients relative to their skeletal muscle involvement. The aim of this study was to assess dysphagia in Duchenne and Becker patients in relation to ambulatory capacity.

How Do Pharmaceutical Companies Model Survival of Cancer Patients? A Review of NICE Single Technology Appraisals in 2017.

Before an intervention is publicly funded within the United Kingdom, the cost-effectiveness is assessed by the National Institute of Health and Care Excellence (NICE). The efficacy of an intervention across the patients' lifetime is often influential of the cost-effectiveness analyses, but is associated with large uncertainties. We reviewed committee documents containing company submissions and evidence review group (ERG) reports to establish the methods used when extrapolating survival data, whether these ...

The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy.

We performed a prospective, cross-sectional cognitive assessment in subjects with Duchenne Muscular Dystrophy (DMD) and their biological mothers.

Guillaume-Benjamin Duchenne: a miserable life dedicated to science.

Duchenne de Boulogne is known mainly by the disease eponymously named "Duchenne muscular dystrophy", or pseudohypertrophic muscular dystrophy, although some experts consider that the original description of this disease does not belong to him. Less well known are the facts related to the tragic death of his beloved wife shortly after the birth of his only child, the unjustifiable distance he was forced to keep from this son for more than 30 years, and of being humiliated and professionally despised by his p...

Molecular and Clinical Characteristics of a National Cohort of Paediatric Duchenne Muscular Dystrophy Patients in Norway.

As new gene-related treatment options for Duchenne muscular dystrophy (DMD) are being developed, precise information about the patients' genetic diagnosis and knowledge about the diversities of natural history in DMD is vital.

A surrogate-based approach for trace organic chemical removal by a high-rejection reverse osmosis membrane.

Public confidence in the safety of recycled water for potable water reuse can be improved by providing assurance regarding high removal of trace organic chemicals (TOrCs) by reverse osmosis (RO) treatment. This pilot-scale study assessed the effectiveness of a surrogate indicator-N-Nitrosodimethlyamine (NDMA)-for ensuring a high level of TOrC removal by a high-rejection RO membrane. The pilot-scale tests showed that the rejection of 23 TOrCs by the high-rejection RO membrane was consistently greater than ND...

Diaphragm Involvement in Duchenne Muscular Dystrophy (DMD): An MRI Study.

Duchenne muscular dystrophy (DMD) is characterized by progressive weakness and wasting of skeletal, cardiac, and respiratory muscles, with consequent cardiopulmonary failure as the main cause of death. Reliable outcome measures able to demonstrate specific trends over disease progression are essential.

An observational study of INR control according to NICE criteria in patients with non-valvular atrial fibrillation-The SAIL Warfarin Out of Range Descriptors Study (SWORDS).

In patients with non-valvular atrial fibrillation (NVAF) prescribed warfarin, the UK National Institute of Health and Care Excellence (NICE) defines poor anticoagulation as a TTR of 

Playing a cooperative game promotes preschoolers' sharing with third-parties, but not social inclusion.

This study examined the effect of gaming context on young children's prosocial behaviors. Dyads of 4- to 5-year-old children (N = 96) played the same game cooperatively, competitively, or solitarily. After playing the game for a total of ten minutes, sharing with and social inclusion of uninvolved third-parties as well as free play with previous co-players was observed. Children shared less with third-parties after playing the game competitively than after playing it cooperatively. Playing a solitary game r...

NICE Guidance - Prostate cancer: diagnosis and management: © NICE (2019) Prostate cancer: diagnosis and management.

Surgical considerations for cardiac allograft rejection.

This article reviews the surgical considerations of cardiac allograft rejection after heart transplantation and describes current treatment modalities for the failing graft. Cardiac allograft rejection can be a moribund diagnosis, especially when it is acute and high grade. It is broadly categorized into hyperacute, acute cellular, and antibody-mediated rejection. Treatment includes a multitude of medical and immunomodulation therapies for graft recovery. Severe rejection requires mechanical circulatory sup...

Can simple and low-cost motor function assessments help in the diagnostic suspicion of Duchenne muscular dystrophy?

Duchenne muscular dystrophy, an X-linked genetic disease, leads to progressive muscle weakness mainly in the lower limbs. Motor function tests help to monitor disease progression. Can low-cost, simple assessments help in the diagnostic suspicion of Duchenne muscular dystrophy? The authors aim to define the sensitivity of time to rise from the floor, time to walk 10meters (10MWT), and time to run 10meters (10MRT), evaluating them as eventual diagnostic screening tools.

The association of acute rejection versus recurrent glomerular disease with graft outcomes after kidney transplantation.

It has been shown that glomerulonephritis (GN) recurrence affects graft survival more than acute rejection. Thus, we assessed allograft survival after biopsy confirmed diagnosis of acute rejection or recurrent GN in current era of immunosuppression.

Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy.

The definition of reliable outcome measures is of increasing interest in patients with Duchenne muscular dystrophy (DMD).

Two-dimensional systolic speckle tracking echocardiography provides a noninvasive aid in the identification of acute pediatric heart transplant rejection.

Acute rejection is an important cause of morbidity and mortality in the pediatric heart transplant (HT) population. A reliable noninvasive method for diagnosis of clinical rejection could substantially reduce these negative outcomes.

Common causes of EID sample rejection in Zimbabwe and how to mitigate them.

Early infant diagnosis (EID) of HIV provides an opportunity for early HIV detection and access to appropriate Antiretroviral treatment (ART). Dried Blood Spot (DBS) samples are used for EID of exposed infants, born to HIV-positive mothers. However, DBS rejection rates in Zimbabwe have been exceeding the target of less than 2% per month set by the National Microbiology Reference Laboratory (NMRL), in Harare. The aim of this study was to determine the DBS sample rejection rate, the reasons for rejection and t...

Are trials of psychological and psychosocial interventions for schizophrenia and psychosis included in the NICE guidelines pragmatic? A systematic review.

The NICE clinical guidelines on psychosocial interventions for the treatment of schizophrenia and psychosis in adults are based on the results of randomized controlled trials (RCTs), which may not be studies with a pragmatic design, leading to uncertainty on applicability or recommendations to everyday clinical practice.

The Risk of Cytomegalovirus Infection after Treatment of Acute Rejection in Renal Transplant Recipients.

The risk of cytomegalovirus infection (CMV) after rejection treatment is poorly understood. To investigate this, we conducted a case/control(1:2) analysis of adult renal-transplant recipients between 1/1/2005-12/31/2015 via incidence-density sampling and survival analysis. Our objective was to evaluate the association of prior acute-rejection with subsequent CMV, including epidemiology and outcomes. There were 2481 eligible renal-transplants within the study period and 251 distinct CMV infections. Despite t...

Digital Therapeutics: Emerging New Therapy for Neurologic Deficits after Stroke.

Digital therapeutics is an evidence-based intervention using high-quality software, with the sole purpose of treatment. As many healthcare systems are encountering high demands of quality outcomes, the need for digital therapeutics is gradually increasing in the clinical field. We conducted review of the implications of digital therapeutics in the treatment of neurological deficits for stroke patients. The implications of digital therapeutics have been discussed in four domains: cognition, speech and aphasi...

Delays in diagnosis of Duchenne muscular dystrophy: an evaluation of genotypic and sociodemographic factors.

This study investigates associations between genotypic and sociodemographic factors and the age of diagnosis of Duchenne muscular dystrophy (DMD).

Detection of lung transplant rejection in a rat model using hyperpolarized 1- C pyruvate-based metabolic imaging.

The current standard for noninvasive imaging of acute rejection consists of X-ray/CT, which derive their contrast from changes in ventilation, inflammation and edema, as well as remodeling during rejection. We propose the use of hyperpolarized [1- C] pyruvate MRI-which provides real-time metabolic assessment of tissue-as an early biomarker for tissue rejection. In this preliminary study, we used μCT-derived parameters and HP C MR-derived biomarkers to predict rejection in an orthotopic left lung transplan...

Haplotype-Based noninvasive prenatal diagnosis for duchenne muscular dystrophy: A pilot study in South China.

To explore the accuracy and feasibility of noninvasive prenatal diagnosis (NIPD) for Duchenne Muscular Dystrophy (DMD) based on the haplotype approach.

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