Zobrazeno 1 - 10
of 10
pro vyhledávání: '"Alexa C. Klimchak"'
Publikováno v:
Health and Quality of Life Outcomes, Vol 22, Iss 1, Pp 1-10 (2024)
Abstract Background Duchenne muscular dystrophy (DMD) is a genetic disease resulting in progressive muscle weakness, loss of ambulation, and cardiorespiratory complications. Direct estimation of health-related quality of life for patients with DMD is
Externí odkaz:
https://doaj.org/article/dc090ed88a654c9989faa8c6a9321700
Autor:
Damon Asher, Daisy Dai, Alexa C. Klimchak, Lauren E. Sedita, Katherine L. Gooch, Louise Rodino-Klapac
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 474-483 (2023)
Gene therapies have potential to improve outcomes of severe diseases after only a single administration. Novel therapies are continually being developed using knowledge gained from prior successes, a concept known as scientific spillover. Gene therap
Externí odkaz:
https://doaj.org/article/69bf28089f5a4ed6a5b08d33b57c34b7
Autor:
Alexa C. Klimchak, Lauren E. Sedita, Louise R. Rodino-Klapac, Jerry R. Mendell, Craig M. McDonald, Katherine L. Gooch, Daniel C. Malone
Publikováno v:
Journal of Market Access & Health Policy, Vol 11, Iss 1 (2023)
ABSTRACTBackground: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by DMD gene mutations. Early cost-effectivenes
Externí odkaz:
https://doaj.org/article/fc618fecb3e6429197793da05d4a6521
Publikováno v:
American Journal of Preventive Cardiology, Vol 5, Iss , Pp 100160- (2021)
Externí odkaz:
https://doaj.org/article/e3ecf1f39a824905952f4b5319644975
Publikováno v:
American Journal of Preventive Cardiology, Vol 1, Iss , Pp 100010- (2020)
Objective: National estimates of atherosclerotic cardiovascular disease (ASCVD) in the United States (US) are scarce, especially for patients grouped by cardiovascular risk, lipid-lowering therapy use, and low-density lipoprotein cholesterol (LDL-C)
Externí odkaz:
https://doaj.org/article/5fe7d7dde0bb45a1835e912aadec6b32
Publikováno v:
Journal of Managed Care & Specialty Pharmacy. 29:472-479
Assessing the value of single or short-term therapies (SSTs) within traditional cost-effectiveness analyses (CEAs) has been a topic of discussion as the number of SSTs increases, particularly regarding the effect of discounting on valuation. To quant
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ae0bb55ae7aa017e10b78a5913926da8
Autor:
Shelagh M. Szabo, Alexa C. Klimchak, Christina Qian, Susan Iannaccone, Evan Popoff, Katherine L. Gooch
Publikováno v:
Journal of neuromuscular diseases. 9(6)
Background: Data on the clinical course of Duchenne muscular dystrophy (DMD) exist from well-characterized clinical cohorts but estimates from real-world populations are fewer. Objective: The objective was to estimate the prevalence of key clinical m
Publikováno v:
American Journal of Preventive Cardiology, Vol 5, Iss, Pp 100160-(2021)
[This corrects the article DOI: 10.1016/j.ajpc.2020.100010.].
Publikováno v:
American Journal of Preventive Cardiology, Vol 1, Iss, Pp 100010-(2020)
Am J Prev Cardiol
American Journal of Preventive Cardiology
American journal of preventive cardiology, vol 1, iss Lancet 376 2010
Am J Prev Cardiol
American Journal of Preventive Cardiology
American journal of preventive cardiology, vol 1, iss Lancet 376 2010
Objective National estimates of atherosclerotic cardiovascular disease (ASCVD) in the United States (US) are scarce, especially for patients grouped by cardiovascular risk, lipid-lowering therapy use, and low-density lipoprotein cholesterol (LDL-C) l