Registries for evaluating patient outcomes: a user's guide
This User's Guide is intended to support the design, implementation, analysis, interpretation,
and quality evaluation of registries created to increase understanding of patient outcomes …
and quality evaluation of registries created to increase understanding of patient outcomes …
[HTML][HTML] Autophagy, lipophagy and lysosomal lipid storage disorders
Autophagy is a catabolic process with an essential function in the maintenance of cellular
and tissue homeostasis. It is primarily recognised for its role in the degradation of …
and tissue homeostasis. It is primarily recognised for its role in the degradation of …
New approaches to Tay-Sachs disease therapy
VV Solovyeva, AA Shaimardanova… - Frontiers in …, 2018 - frontiersin.org
Tay-Sachs disease belongs to the group of autosomal-recessive lysosomal storage
metabolic disorders. This disease is caused by β-hexosaminidase A (HexA) enzyme …
metabolic disorders. This disease is caused by β-hexosaminidase A (HexA) enzyme …
Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
W Hughes-Wilson, A Palma, A Schuurman… - Orphanet journal of rare …, 2012 - Springer
Since its enactment in 2000, the European Orphan Medicinal Products Regulation has
allowed the review and approval of approaching 70 treatments for some 55 different …
allowed the review and approval of approaching 70 treatments for some 55 different …
Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease
A Zimran, E Brill-Almon, R Chertkoff… - Blood, The Journal …, 2011 - ashpublications.org
Abstract Taliglucerase alfa (Protalix Biotherapeutics, Carmiel, Israel) is a novel plant cell–
derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 3, double …
derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 3, double …
Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Acquired thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease
characterized by thrombotic microangiopathy leading to end-organ damage. The standard of …
characterized by thrombotic microangiopathy leading to end-organ damage. The standard of …
Glucocerebrosidase mutations and synucleinopathies: toward a model of precision medicine
Glucocerebrosidase is a lysosomal enzyme. The characterization of a direct link between
mutations in the gene coding for glucocerebrosidase (GBA1) with the development of …
mutations in the gene coding for glucocerebrosidase (GBA1) with the development of …
[HTML][HTML] The clinical decision analysis using decision tree
JM Bae - Epidemiology and health, 2014 - ncbi.nlm.nih.gov
The clinical decision analysis (CDA) has used to overcome complexity and uncertainty in
medical problems. The CDA is a tool allowing decision-makers to apply evidence-based …
medical problems. The CDA is a tool allowing decision-makers to apply evidence-based …
Economic costs associated with acute attacks and long-term management of hereditary angioedema
DA Wilson, K Bork, EP Shea, AM Rentz… - Annals of Allergy …, 2010 - Elsevier
BACKGROUND: Hereditary angioedema (HAE) is a rare autosomal dominant disorder
characterized by recurrent acute attacks of swelling of the larynx, abdomen, and periphery …
characterized by recurrent acute attacks of swelling of the larynx, abdomen, and periphery …
[图书][B] Just caring: health care rationing and democratic deliberation
LM Fleck - 2009 - books.google.com
What does it mean to be a" just" and" caring" society when we have only limited resources to
meet unlimited health care needs? Do we believe that all lives are of equal value? Is human …
meet unlimited health care needs? Do we believe that all lives are of equal value? Is human …