Mobility and Quality of Life in Adults with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement
Asfotase alfa treatment improved mobility, physical function, pain, and HRQoL and was well tolerated. These data show the benefit of asfotase alfa in adults with paediatric-onset HPP.
Authors
- Tarekegn Geberhiwot (ORCID: https://orcid.org/0000-0002-3629-2338)
- Jennifer Walsh (ORCID: https://orcid.org/0000-0002-7122-2650)
- Judith Bubbear (ORCID: https://orcid.org/0000-0001-7576-250X)
- Kenneth Poole (ORCID: https://orcid.org/0000-0002-9187-9967)
- Richard Keen (ORCID: https://orcid.org/0009-0001-8216-3828)
- M K Javaid (ORCID: https://orcid.org/0000-0001-7985-0048)
- Peter Selby (ORCID: https://orcid.org/0000-0001-9465-9268)
- Katie Moss (ORCID: https://orcid.org/0000-0002-1944-8302)
- Shona Fang (ORCID: https://orcid.org/0009-0008-7019-5647)
- Alexandros Zygouras
Institutions
- St George's Hospital (GB)
- University of Cambridge (GB)
- Queen Elizabeth Hospital Birmingham (GB)
- St George’s University Hospitals NHS Foundation Trust (GB)
- Manchester Royal Infirmary (GB)
- Royal National Orthopaedic Hospital (GB)
- National Institute for Health and Care Research (GB)
- University of Oxford (GB)
- St George's Hospital (GB)
- Alexion Pharmaceuticals (United States) (US)
- AstraZeneca (United States) (US)
- University College London (GB)
- Alexion Pharma (Switzerland) (CH)
- University of Sheffield (GB)
Publication Details
- Journal
- Advances in Therapy
- Published
- 2025-03-26
- DOI
- https://doi.org/10.1007/s12325-025-03168-w
- Citations
- 4
- Primary Topic
- Alkaline Phosphatase Research Studies
- Type
- article
- Field-Weighted Citation Impact
- 5.18
Funders
- AstraZeneca
- Alexion Pharmaceuticals
- National Institute for Health and Care Research
- NIHR Cambridge Biomedical Research Centre