Detalhe da pesquisa
1.
Phase I study of liver depot gene therapy in late-onset Pompe disease.
Mol Ther
; 31(7): 1994-2004, 2023 07 05.
Artigo
Inglês
| MEDLINE | ID: mdl-36805083
2.
Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease.
Mol Genet Metab
; 129(2): 67-72, 2020 02.
Artigo
Inglês
| MEDLINE | ID: mdl-31839530
3.
Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease.
Mol Ther
; 26(9): 2304-2314, 2018 09 05.
Artigo
Inglês
| MEDLINE | ID: mdl-30025991
4.
Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
Mol Genet Metab
; 123(2): 92-96, 2018 02.
Artigo
Inglês
| MEDLINE | ID: mdl-29289479
5.
High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient.
Mol Genet Metab
; 122(1-2): 76-79, 2017 09.
Artigo
Inglês
| MEDLINE | ID: mdl-28648664
6.
Screening data from 19 patients with late-onset Pompe disease for a phase I clinical trial of AAV8 vector-mediated gene therapy.
JIMD Rep
; 64(5): 393-400, 2023 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-37701327