Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey

INTRODUCTION: Onasemnogene abeparvovec (OA) is the only gene replacement therapy currently approved for spinal muscular atrophy (SMA) treatment. We sought to assess real-world patient and caregiver outcomes after OA treatment for SMA. METHODS: Patients who received OA were identified from the 2021 C...

Descripción completa

Detalles Bibliográficos
Autores principales: Toro, Walter, Yang, Min, Georgieva, Mihaela, Anderson, Annika, LaMarca, Nicole, Patel, Anish, Akbarnejad, Hanane, Dabbous, Omar
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2023
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10611830/
https://www.ncbi.nlm.nih.gov/pubmed/37776479
http://dx.doi.org/10.1007/s12325-023-02685-w

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10611830/
https://www.ncbi.nlm.nih.gov/pubmed/37776479
http://dx.doi.org/10.1007/s12325-023-02685-w

Ejemplares similares