Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Simon Shohet"'
Autor:
Kristl G. Claeys, Hani Kushlaf, Syed Raza, Noemi Hummel, Simon Shohet, Ian Keyzor, Agnieszka Kopiec, Ryan Graham, Brian Fox, Benedikt Schoser
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-10 (2024)
Abstract Background The minimal clinically important difference (MCID) is the smallest change in outcome that physicians or patients would consider meaningful and is relevant when evaluating disease progression or the efficacy of interventions. Studi
Externí odkaz:
https://doaj.org/article/038589708d914771b4227aa70fd5c9b8
Autor:
Priya S. Kishnani, Simon Shohet, Syed Raza, Noemi Hummel, Jeffrey P. Castelli, Sheela Sitaraman Das, Heng Jiang, Agnieszka Kopiec, Ian Keyzor, Andreas Hahn
Publikováno v:
Journal of Patient-Reported Outcomes, Vol 8, Iss 1, Pp 1-12 (2024)
Abstract Background The construct validity and interpretation of the Patient-Reported Outcome Measurement Information System (PROMIS®) Physical Function short form 20a (PF20a) questionnaire were evaluated for patients with late-onset Pompe disease (
Externí odkaz:
https://doaj.org/article/2bea6ae7ace94f38add7a9b92b63452c
Autor:
Priya Kishnani, Barry Byrne, Kristl Claeys, Paula Clemens, Jordi Díaz-Manera, Mazen Dimachkie, Hani Kushlaf, Tahseen Mozaffar, Mark Roberts, Antonio Toscano, Noemi Hummel, Fred Holdbrook, Simon Shohet, Benedikt Schoser
Publikováno v:
Genetics in Medicine Open, Vol 2, Iss , Pp 100905- (2024)
Externí odkaz:
https://doaj.org/article/7e8f3ffae6b041ad994f1232138e95db
Autor:
Ian Keyzor, Simon Shohet, Jeff Castelli, Sheela Sitaraman, Biliana Veleva-Rotse, Jill M. Weimer, Brian Fox, Tobias Willer, Steve Tuske, Louise Crathorne, Klara J. Belzar
Publikováno v:
Biomolecules, Vol 13, Iss 8, p 1227 (2023)
The treatment landscape for lysosomal storage disorders (LSDs) is rapidly evolving. An increase in the number of preclinical and clinical studies in the last decade has demonstrated that pharmacological chaperones are a feasible alternative to enzyme
Externí odkaz:
https://doaj.org/article/77e7ce7c2b764f51880d261188c06a9c
Autor:
Jeff Castelli, Shuai Fu, Noemi Hummel, Tahseen Mozaffar, Brian Fox, Ian Keyzor, Syed Raza, Sheela Sitaraman, Simon Shohet
Publikováno v:
Molecular Genetics and Metabolism. 138:107046
Publikováno v:
International Journal of Technology Assessment in Health Care. 38:S84-S84
IntroductionPompe disease (PD) is a rare, progressive neuromuscular disease that severely affects motor and respiratory functions. Late onset PD (LOPD) is the most common phenotype. Current treatment involves enzyme replacement therapy (ERT) with alg
Autor:
Gemma Wood, Simon Shohet
Publikováno v:
Journal of Commercial Biotechnology. 9:59-66
As the list of biological drugs gaining regulatory approval grows, this paper examines how different technological approaches to delivering these drugs have evolved, and how business strategies have developed to meet the challenges. Companies can bro
Autor:
Simon Shohet
Publikováno v:
Journal of Commercial Biotechnology. 12:5-7
Autor:
Simon Shohet, Martha Prevezer
Publikováno v:
R&D Management. 26:283-298
One prominent feature of biotechnology in the UK is the close relationship between the public-sector science base and industry. Using information from interviews, the system for technology transfer is examined and four main institutional actors are i
Autor:
Simon Shohet
Publikováno v:
Science and Public Policy. 23:117-122
The technical arguments for and against process-based regulation of biotechnology are reviewed, and the positions of proponents and opponents are examined, using the UK as a case. In particular, the findings of the Royal Commission on Environmental P