Detalhe da pesquisa
1.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34788506
2.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35065785
3.
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Lancet
; 388(10043): 476-87, 2016 Jul 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-27289174
4.
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.
Hepatology
; 68(6): 2434-2437, 2018 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30014500
5.
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Nat Med
; 30(2): 488-497, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-38355973
6.
How to START? Four pillars to optimally begin your orphan drug development.
Orphanet J Rare Dis
; 18(1): 229, 2023 08 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37537670
7.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Nat Med
; 25(2): 234-241, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30664781
8.
Defining rare conditions in the era of personalized medicine.
Nat Rev Drug Discov
; 22(11): 857-858, 2023 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37684343
9.
IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases.
Nat Rev Drug Discov
; 22(12): 937-938, 2023 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37872324
10.
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.
Orphanet J Rare Dis
; 13(1): 49, 2018 04 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29625577
11.
The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy.
Hemasphere
; 6(2): e671, 2022 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-35198856
12.
Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook.
Nat Rev Drug Discov
; 19(8): 495-496, 2020 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-32313251