Mobility and Quality of Life in Children with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement
This analysis confirmed the clinical benefit of asfotase alfa in children with HPP. Asfotase alfa was well tolerated, with no new safety signals identified.
Authors
- Paul Arundel (ORCID: https://orcid.org/0000-0001-8443-3601)
- Vrinda Saraff (ORCID: https://orcid.org/0000-0003-3601-8942)
- Nick Bishop (ORCID: https://orcid.org/0000-0001-7263-8546)
- Zulf Mughal (ORCID: https://orcid.org/0000-0002-8603-5663)
- Raja Padidela (ORCID: https://orcid.org/0000-0002-3350-9896)
- Nick J. Shaw (ORCID: https://orcid.org/0000-0001-8302-4144)
- Shona Fang (ORCID: https://orcid.org/0009-0008-7019-5647)
- Alexandros Zygouras
Institutions
- Manchester Academic Health Science Centre (GB)
- Alexion Pharmaceuticals (United States) (US)
- AstraZeneca (United States) (US)
- Royal Manchester Children's Hospital (GB)
- Sheffield Children's Hospital (GB)
- University of Birmingham (GB)
- University of Sheffield (GB)
Publication Details
- Journal
- Advances in Therapy
- Published
- 2025-05-29
- DOI
- https://doi.org/10.1007/s12325-025-03225-4
- Citations
- 1
- Primary Topic
- Alkaline Phosphatase Research Studies
- Type
- article
- Field-Weighted Citation Impact
- 1.29
Funders
- AstraZeneca
- Alexion Pharmaceuticals