Enzyme replacement therapy for late‐onset Pompe disease
Información
- DOI:
- https://doi.org/10.1002/14651858.CD012993Copiar DOI
- Base de datos:
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- Cochrane Database of Systematic Reviews
- Versión publicada:
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- 20 abril 2018see what's new
- Tipo:
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- Intervention
- Etapa:
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- Protocol
- Grupo Editorial Cochrane:
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Grupo Cochrane de Fibrosis quística y enfermedades genéticas
- Copyright:
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- Copyright © 2018 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd.
Cifras del artículo
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Autores
Contributions of authors
Protocol stage: draft or comment on the protocol (or both): all authors.
Sources of support
Internal sources
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No sources of support supplied
External sources
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National Institute for Health Research, UK.
This systematic review was supported by the National Institute for Health Research, via Cochrane Infrastructure funding to the Cochrane Cystic Fibrosis and Genetic Disorders Group.
Declarations of interest
Reena Sharma: none known.
Uma Ramaswami: none known.
Duncan Cole: has received honoraria and consultancy fees from Genzyme, and is in receipt of a grant for service support from Shire.
Mark Roberts: has received payment from Genzyme, Amicus and Biomarin for attendance on Medical Advisory Boards and for travel costs for attendance at conferences. Payment received from Genzyme for lectures on Pompe disease.
Christian Hendriksz: owns his medical education company FYMCA Medical Ltd , provides consultancy services to the pharmaceutical industry, health care providers, government organisations, patient organisations and regulators in both his personal capacity as well as for his institution.
Karolina Stepien: none known.
Derralynn Hughes: has received consultancy fees, shared with her institution and speakers fees from Genzyme and Biomarin for projects in relation to other storage disorders and my institution has received grants for clinical research trials from Biomarin in relation to Pompe disease and from Genzyme for other storage disorders.
Ashma Krishan: none known.
Nikki Jahnke: none known.
Acknowledgements
This protocol was developed in collaboration with Cochrane Neuromuscular. The editorial process was primarily managed by the Cochrane Cystic Fibrosis and Genetic Disorders Group and the editorial team from Cochrane Neuromuscular provided comments and peer review at both title proposal and protocol stages.
We thank Natalie Hall, Information Specialist from the Cochrane Cystic Fibrosis and Genetic Disorders Group, for her assistance with developing the search strategies.
This project was supported by the National Institute for Health Research, via Cochrane Infrastructure funding to the Cochrane Cystic Fibrosis and Genetic Disorders Group and Cochrane Neuromuscular. The views and opinions expressed therein are those of the authors and do not necessarily reflect those of the Systematic Reviews Programme, NIHR, NHS or the Department of Health.
Version history
| Published | Title | Stage | Authors | Version |
| 2023 Dec 12 | Enzyme replacement therapy for late‐onset Pompe disease | Review | Sanjush Dalmia, Reena Sharma, Uma Ramaswami, Derralynn Hughes, Nikki Jahnke, Duncan Cole, Sherie Smith, Tracey Remmington | |
| 2018 Apr 20 | Enzyme replacement therapy for late‐onset Pompe disease | Protocol | Reena Sharma, Derralynn Hughes, Uma Ramaswami, Duncan Cole, Mark Roberts, Christian J Hendriksz, Karolina M Stepien, Ashma Krishan, Nikki Jahnke | |
