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Page 1
Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity.
Vallée TC, Glasmacher JS, Buchner H, Arkwright PD, Behrends U, Bondarenko A, Browning MJ, Buchbinder DK, Cattoni A, Chernyshova L, Ciznar P, Cole T, Czogala W, Dueckers G, Edgar JDM, Erbey F, Fasth A, Ferrua F, Formankova R, Gambineri E, Gennery AR, Goldman FD, Gonzalez-Granado LI, Heilmann C, Heiskanen-Kosma T, Juntti H, Kainulainen L, Kanegane H, Karaca NE, Kilic SS, Klein C, Koltan S MD, PhD, Kondratenko I, Meyts I, Nasrullayeva GM, Notarangelo LD, Pasic S, Pellier I, Pignata C, Misbah SA, Schulz AS, Segundo GR, Shcherbina A, Slatter MA, Sokolic R, Soler-Palacin P, Stepensky P, van Montfrans JM, Ryhänen S, Wolska-Kuśnierz B MD, PhD, Ziegler JB, Zhao X, Aiuti A, Ochs HD, Albert MH. Vallée TC, et al. Among authors: cattoni a. Blood. 2024 Apr 5:blood.2023021411. doi: 10.1182/blood.2023021411. Online ahead of print. Blood. 2024. PMID: 38579284
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: cattoni a. 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
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, Gasperini S, Galimberti S, Silvani P, Filisetti C, Darin S, Forni G, Miglietta S, Santi L, Facchini M, Corti A, Fumagalli F, Cicalese MP, Calbi V, Migliavacca M, Barzaghi F, Ferrua F, Gallo V, Recupero S, Canarutto D, Doglio M, Tedesco L, Volpi N, Rovelli A, la Marca G, Valsecchi MG, Zancan S, Ciceri F, Naldini L, Baldoli C, Parini R, Gentner B, Aiuti A, Bernardo ME. Consiglieri G, et al. Among authors: cattoni a. Sci Transl Med. 2024 May;16(745):eadi8214. doi: 10.1126/scitranslmed.adi8214. Epub 2024 May 1. Sci Transl Med. 2024. PMID: 38691622 Clinical Trial.
Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multi-center nation-wide cohort.
Casale M, Forni GL, Cassinerio E, Pasquali D, Origa R, Serra M, Campisi S, Peluso A, Renni R, Cattoni A, De Michele E, Allò M, Poggi M, Ferrara F, Di Concilio R, Sportelli F, Quarta A, Putti MC, Notarangelo LD, Sau A, Ladogana S, Tartaglione I, Picariello S, Marcon A, Sturiale P, Roberti D, Lazzarino AI, Perrotta S. Casale M, et al. Among authors: cattoni a. Haematologica. 2022 Feb 1;107(2):467-477. doi: 10.3324/haematol.2020.272419. Haematologica. 2022. PMID: 33406815 Free PMC article.
SMC1A epilepsy syndrome: clinical data from a large international cohort.
Gibellato E, Cianci P, Mariani M, Parma B, Huisman S, Śmigiel R, Bisgaard AM, Massa V, Gervasini C, Moretti A, Cattoni A, Biondi A, Selicorni A. Gibellato E, et al. Among authors: cattoni a. Am J Med Genet A. 2024 Feb 29:e63577. doi: 10.1002/ajmg.a.63577. Online ahead of print. Am J Med Genet A. 2024. PMID: 38421079
Mucopolysaccharidosis-Plus Syndrome, a Rapidly Progressive Disease: Favorable Impact of a Very Prolonged Steroid Treatment on the Clinical Course in a Child.
Faraguna MC, Musto F, Crescitelli V, Iascone M, Spaccini L, Tonduti D, Fedeli T, Kullmann G, Canonico F, Cattoni A, Dell'Acqua F, Rizzari C, Gasperini S. Faraguna MC, et al. Among authors: cattoni a. Genes (Basel). 2022 Feb 28;13(3):442. doi: 10.3390/genes13030442. Genes (Basel). 2022. PMID: 35327996 Free PMC article. Review.
Long-Term Complications after Allogeneic Hematopoietic Stem Cell Transplantation with Treosulfan- or Busulfan-Based Conditioning in Pediatric Patients with Acute Leukemia or Myelodysplastic Syndrome: Results of an Associazione Italiana Ematologia Oncologia Pediatrica Retrospective Study.
Saglio F, Pagliara D, Zecca M, Balduzzi A, Cattoni A, Prete A, Tambaro FP, Faraci M, Calore E, Locatelli F, Fagioli F; AIEOP BMT Group. Saglio F, et al. Among authors: cattoni a. Transplant Cell Ther. 2024 Apr;30(4):433.e1-433.e10. doi: 10.1016/j.jtct.2023.12.671. Epub 2024 Jan 2. Transplant Cell Ther. 2024. PMID: 38176654
Endocrine, auxological and metabolic profile in children and adolescents with Down syndrome: from infancy to the first steps into adult life.
Molinari S, Fossati C, Nicolosi ML, Di Marco S, Faraguna MC, Limido F, Ocello L, Pellegrinelli C, Lattuada M, Gazzarri A, Lazzerotti A, Sala D, Vimercati C, Capitoli G, Daolio C, Biondi A, Balduzzi A, Cattoni A. Molinari S, et al. Among authors: cattoni a. Front Endocrinol (Lausanne). 2024 Apr 8;15:1348397. doi: 10.3389/fendo.2024.1348397. eCollection 2024. Front Endocrinol (Lausanne). 2024. PMID: 38654931 Free PMC article. Review.
68 results