Delayed Infusion Reactions to Enzyme Replacement Therapies [PDF]
, 2016 There are currently ten intravenous enzyme replacement therapy (ERT) products available for the treatment of eight different lysosomal diseases (LD) in the USA. Additional ERT products are in clinical trials. The most common ERT adverse events are infusion reactions (IR).
Zahra, Karimian +3 more
openaire +2 more sources
CCDC80 suppresses high‐grade serous ovarian cancer migration via negative regulation of B7‐H3
Molecular Oncology, EarlyView.PAX8 is a lineage‐specific master regulator of transcription in high‐grade serous ovarian cancer (HGSC) progression. We show for the first time that PAX8 facilitates proliferation and metastasis by repressing the cell autonomous tumor suppressor CCDC80 and inducing B7‐H3 expression.
Aya Saleh +12 more
wiley +1 more source
No Difference in Health Related Quality of Life Between Therapeutic Options for Type 1 Gaucher Disease [PDF]
, 2016 Type 1 Gaucher disease (GD) is the most common lysosomal storage disorder. Previously, treatment for GD was limited to intravenous enzyme replacement therapy (ERT).
Wagner, Victoria
core +1 more source
Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H. [PDF]
, 2016 Mucopolysaccharidosis (MPS) type-IH is a lysosomal storage disease that results from mutations in the IDUA gene causing the accumulation of glycosaminoglycans (GAGs).
Dickson, Patricia I +6 more
core +1 more source
Overview of molecular signatures of senescence and associated resources: pros and cons
FEBS Open Bio, EarlyView.Cells can enter a stress response state termed cellular senescence that is involved in various diseases and aging. Detecting these cells is challenging due to the lack of universal biomarkers. This review presents the current state of senescence identification, from biomarkers to molecular signatures, compares tools and approaches, and highlights ...
Orestis A. Ntintas +6 more
wiley +1 more source
Cell migration and chimerism after whole‐organ transplantation: The basis of graft acceptance [PDF]
, 1993 Improvements in the prevention or control of rejection of the kidney and liver have been largely interchangeable ( 1 , 2 ) and then applicable, with very little modification, to thoracic and other organs.
Demetris, AJ +13 more
core +1 more source
Enzyme replacement therapy: conception, chaos and culmination [PDF]
Philosophical Transactions of the Royal Society of London. Series B: Biological Sciences, 2003 Soon after the enzymatic defects in Gaucher disease and in Niemann–Pick disease were discovered, enzyme replacement or enzyme supplementation was proposed as specific treatment for patients with these and related metabolic storage disorders. While relatively straightforward in concept, successful implementation of this approach required many years of ...
openaire +2 more sources
FEBS Open Bio, EarlyView.We investigated the toxicity of 12 active compounds commonly found in herbal weight loss supplements (WLS) using human liver and colon cell models. Epigallocatechin‐3‐gallate was the only compound showing significant toxicity. Metabolic profiling revealed protein degradation, disrupted energy and lipid metabolism suggesting that the inclusion of EGCG ...
Emily C. Davies +3 more
wiley +1 more source
Hypophosphatasia in childhood: Diagnosis to management
Osteoporosis and SarcopeniaHypophosphatasia (HPP) is a rare inherited metabolic bone disorder caused by loss-of-function mutations in the ALPL gene, leading to deficient activity of tissue-nonspecific alkaline phosphatase (TNSALP).
Minji Im, Sung Yoon Cho
doaj +1 more source
Presenting signs and patient co-variables in Gaucher disease : outcome of the Gaucher Earlier Diagnosis Consensus (GED-C) Delphi initiative [PDF]
, 2019 © 2018 The Authors. Internal Medicine Journal by Wiley Publishing Asia Pty Ltd on behalf of Royal Australasian College of Physicians.Background: Gaucher disease (GD) presents with a range of signs and symptoms.
Belmatoug , N +24 more
core +6 more sources