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Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.
Pontesilli S, Baldoli C, Rosa PAD, Cattoni A, Bernardo ME, Meregalli P, Gasperini S, Motta S, Fumagalli F, Tucci F, Baciga F, Consiglieri G, Canonico F, De Lorenzo P, Chiapparini L, Gentner B, Aiuti A, Biondi A, Rovelli A, Parini R. Pontesilli S, et al. Among authors: bernardo me. J Pediatr. 2022 Jan;240:297-301.e5. doi: 10.1016/j.jpeds.2021.09.020. Epub 2021 Sep 20. J Pediatr. 2022. PMID: 34547335
Efficacy of two different doses of rabbit anti-T-lymphocyte globulin to prevent graft-versus-host disease in children with haematological malignancies transplanted from an unrelated donor: a multicentre, randomised, open-label, phase 3 trial.
Locatelli F, Bernardo ME, Bertaina A, Rognoni C, Comoli P, Rovelli A, Pession A, Fagioli F, Favre C, Lanino E, Giorgiani G, Merli P, Pagliara D, Prete A, Zecca M. Locatelli F, et al. Among authors: bernardo me. Lancet Oncol. 2017 Aug;18(8):1126-1136. doi: 10.1016/S1470-2045(17)30417-5. Epub 2017 Jul 10. Lancet Oncol. 2017. PMID: 28705454 Free article. Clinical Trial.
Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I.
Azario I, Pievani A, Del Priore F, Antolini L, Santi L, Corsi A, Cardinale L, Sawamoto K, Kubaski F, Gentner B, Bernardo ME, Valsecchi MG, Riminucci M, Tomatsu S, Aiuti A, Biondi A, Serafini M. Azario I, et al. Among authors: bernardo me. Sci Rep. 2017 Aug 25;7(1):9473. doi: 10.1038/s41598-017-09958-9. Sci Rep. 2017. PMID: 28842642 Free PMC article.
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini G, Chiesa R, Arora R, Ciotti F, Sarzana M, Antonioli G, Baldoli C, Morena F, Martino S, Ardissino GL, Sora MGN, Naldini L, Ciceri F, Aiuti A, Bernardo ME. Calbi V, et al. Among authors: bernardo me. Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29. Bone Marrow Transplant. 2018. PMID: 29379168 Free PMC article. No abstract available.
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.
Tucci F, Calbi V, Barzaghi F, Migliavacca M, Ferrua F, Bernardo ME, Canarutto D, Consiglieri G, Recupero S, Calzatini F, Gabaldo M, Lucano C, Casiraghi M, Darin S, Dionisio F, Marktel S, Cestone E, Finazzi R, Mieli-Vergani G, Boeri E, Appleby J, Abd Elaziz D, Ciceri F, Aiuti A, Cicalese MP. Tucci F, et al. Among authors: bernardo me. Hepatology. 2018 Dec;68(6):2434-2437. doi: 10.1002/hep.30160. Epub 2018 Nov 13. Hepatology. 2018. PMID: 30014500 Free PMC article. No abstract available.
Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.
Fraldi A, Serafini M, Sorrentino NC, Gentner B, Aiuti A, Bernardo ME. Fraldi A, et al. Among authors: bernardo me. Ital J Pediatr. 2018 Nov 16;44(Suppl 2):130. doi: 10.1186/s13052-018-0565-y. Ital J Pediatr. 2018. PMID: 30442177 Free PMC article. Review.
133 results