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Impact of the COVID-19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatment
BACKGROUND: Cerliponase alfa, an enzyme replacement therapy for neuronal ceroid lipofuscinosis type 2 (CLN2), is currently available in England through a managed access agreement (MAA). It is administered every 2 weeks via an intracerebroventricular device. Here we report qualitative research with f...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8767529/ https://www.ncbi.nlm.nih.gov/pubmed/35045884 http://dx.doi.org/10.1186/s13023-021-02147-y |