Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. [PDF]
, 2016 Taliglucerase alfa is the first available plant cell-expressed human recombinant therapeutic protein. It is indicated for treatment of patients with type 1 Gaucher disease (GD) in adult and pediatric patients in several countries.
Amato, Dominick J +9 more
core +2 more sources
The prevalence of sarcopenia and its effect on prognosis in patients with Crohn’s disease [PDF]
Intestinal Research, 2020 Background/Aims Crohn’s disease is associated with altered body composition, such as low muscle mass, which can affect clinical outcomes. However, there are few studies regarding the effect of sarcopenia on prognosis of Crohn’s disease. In this study, we
Chan Hyung Lee +9 more
doaj +1 more source
Association Between Blood Pressure and Adverse Renal Events in Type 1 Diabetes. [PDF]
, 2016 ObjectiveTo compare different blood pressure (BP) levels in their association with the risk of renal outcomes in type 1 diabetes and to determine whether an intensive glycemic control strategy modifies this association.Research design and methodsWe ...
Gitelman, Stephen E +5 more
core +2 more sources
CETP gene polymorphisms and haplotypes are explanatory variables for HDL cholesterol level in sickle cell disease [PDF]
Brazilian Journal of Medical and Biological ResearchVariations in lipid profile have been observed in sickle cell disease (SCD) and understanding their relationship with disease severity is crucial. This study aimed to investigate the association of polymorphisms of the CETP gene and laboratory markers of
N.R.C. Cruz +8 more
doaj +1 more source
The laboratory findings and different COVID-19 severities: a systematic review and meta-analysis
Annals of Clinical Microbiology and Antimicrobials, 2021 Background Abnormal laboratory findings are common in patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). The aim of this systematic review was to investigate the effect of the level of some laboratory factors (C-reactive
Erfan Kazemi +3 more
doaj +1 more source
Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients. [PDF]
, 2015 Quantification of disease-associated proteins in the cerebrospinal fluid (CSF) has been critical for the study and treatment of several neurodegenerative disorders; however, mutant huntingtin protein (mHTT), the cause of Huntington's disease (HD), is at ...
Andreas Weiss +16 more
core +1 more source
Psychosocial Outcomes in Patients With Endocrine Tumor Syndromes: A Systematic Review
Pediatric Blood &Cancer, EarlyView.ABSTRACT Introduction The combination of disease manifestations, the familial burden, and varying penetrance of endocrine tumor syndromes (ETSs) is unique. This review aimed to portray and summarize available data on psychosocial outcomes in patients with ETSs and explore gaps and opportunities for future research and care.
Daniël Zwerus +6 more
wiley +1 more source
The Laboratory-Based Intermountain Validated Exacerbation (LIVE) Score Identifies Chronic Obstructive Pulmonary Disease Patients at High Mortality Risk. [PDF]
, 2018 Background: Identifying COPD patients at high risk for mortality or healthcare utilization remains a challenge. A robust system for identifying high-risk COPD patients using Electronic Health Record (EHR) data would empower targeting interventions aimed ...
Arjomandi, Mehrdad +9 more
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Cyclophosphamide (CY) is associated with potentially fatal cardiotoxicity, yet no electrocardiographic indices have been established for early detection of CY‐induced cardiomyopathy. This study aimed to determine whether corrected QT interval (QTc) prolongation can predict early onset of CY‐related cardiac dysfunction in pediatric ...
Junpei Kawamura +5 more
wiley +1 more source
Advances in Hematology, 2020 Background. It is estimated that one out of every three Ghanaians has hemoglobin genotype mutation. This change in genetic make-up may result in genotypes such as HbAS, HbSS, and HbSC.
Daniel Kpodji Awaitey +7 more
doaj +1 more source