Detalhe da pesquisa
1.
Long-term Efficacy and Safety of Elamipretide in Patients with Barth Syndrome: 168-Week Open-label Extension Results of TAZPOWER.
Genet Med
; : 101138, 2024 Apr 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-38602181
2.
A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.
Genet Med
; 23(3): 471-478, 2021 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33077895
3.
Quality of life in Barth syndrome.
Ther Adv Rare Dis
; 3: 26330040221093743, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-37180415
4.
Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome.
Orphanet J Rare Dis
; 17(1): 336, 2022 09 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36056411