Detalhe da pesquisa
1.
TISBE: A Public Web Platform for the Consensus-Based Explainable Prediction of Developmental Toxicity.
Chem Res Toxicol
; 37(2): 323-339, 2024 02 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-38200616
2.
Development of a Thalassemia International Prognostic Scoring System (TIPSS).
Blood Cells Mol Dis
; 99: 102710, 2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36463683
3.
Primary HBB gene mutation severity and long-term outcomes in a global cohort of ß-thalassaemia.
Br J Haematol
; 196(2): 414-423, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34697800
4.
Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project.
Br J Clin Pharmacol
; 88(12): 5052-5059, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-34931373
5.
Overview of the European post-authorisation study register post-authorization studies performed in Europe from September 2010 to December 2018.
Pharmacoepidemiol Drug Saf
; 31(6): 689-705, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35092329
6.
A complication risk score to evaluate clinical severity of thalassaemia syndromes.
Br J Haematol
; 192(3): 626-633, 2021 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33216983
7.
Survey by TEDDY European Network of Excellence for Paediatric Clinical Research demonstrates potential for Europe-wide trials.
Acta Paediatr
; 109(3): 607-612, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31532836
8.
Machine Learning Systems Applied to Health Data and System.
Eur J Health Law
; 27(3): 242-258, 2020 05 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-33652397
9.
Risk of mortality from anemia and iron overload in nontransfusion-dependent ß-thalassemia.
Am J Hematol
; 97(2): E78-E80, 2022 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34862982
10.
The ethical framework for performing research with rare inherited neurometabolic disease patients.
Eur J Pediatr
; 176(3): 395-405, 2017 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-28093642
11.
Survival and causes of death in 2,033 patients with non-transfusion-dependent ß-thalassemia
Haematologica
; 106(9): 2489-2492, 2021 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33882642
12.
The International Hemoglobinopathy Research Network (INHERENT): An international initiative to study the role of genetic modifiers in hemoglobinopathies.
Am J Hematol
; 96(11): E416-E420, 2021 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34406671
13.
Special Issue "Personalized Medicine in Blood Disease of Children".
J Pers Med
; 14(3)2024 Mar 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-38541027
14.
Target therapy for high-risk neuroblastoma treatment: integration of regulatory and scientific tools is needed.
Front Med (Lausanne)
; 10: 1113460, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37521350
15.
Iron chelation therapy in thalassemia major: a systematic review with meta-analyses of 1520 patients included on randomized clinical trials.
Blood Cells Mol Dis
; 47(3): 166-75, 2011 Oct 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21843958
16.
Paediatric Medicines in Europe: The Paediatric Regulation-Is It Time for Reform?
Front Med (Lausanne)
; 8: 593281, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-33604345
17.
The conect4children (c4c) Consortium: Potential for Improving European Clinical Research into Medicines for Children.
Pharmaceut Med
; 35(2): 71-79, 2021 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33539007
18.
Medical Device Development for Children and Young People-Reviewing the Challenges and Opportunities.
Pharmaceutics
; 13(12)2021 Dec 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-34959459
19.
The Task-force in Europe for Drug Development for the Young (TEDDY) Network of Excellence.
Paediatr Drugs
; 11(1): 18-21, 2009.
Artigo
em Inglês
| MEDLINE | ID: mdl-19127946
20.
Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
Orphanet J Rare Dis
; 12(1): 64, 2017 04 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28372595