Detalhe da pesquisa
1.
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
Mol Genet Genomic Med
; 9(5): e1664, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33755338
2.
Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.
PLoS One
; 14(5): e0213649, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-31042754
3.
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Orphanet J Rare Dis
; 14(1): 102, 2019 05 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31072340