Nome |
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Adrenal insufficiency: An emerging challenge in thalassemia?, file dfa8b9a0-e6d5-748b-e053-3a05fe0a3a96
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239
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Liver steatosis is highly prevalent and is associated with metabolic risk factors and liver fibrosis in adult patients with type 1 Gaucher disease, file dfa8b9a4-9387-748b-e053-3a05fe0a3a96
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233
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SARS-CoV-2 infection in beta thalassemia : preliminary data from the Italian experience, file dfa8b9a1-bd91-748b-e053-3a05fe0a3a96
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227
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Elevated liver iron concentration is a marker of increased morbidity in patients with beta thalassemia intermedia, file dfa8b99d-e602-748b-e053-3a05fe0a3a96
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209
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Activin receptor-ligand trap for the treatment of β-thalassemia : a serendipitous discovery, file dfa8b9a4-93f6-748b-e053-3a05fe0a3a96
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204
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Innovative Treatments for Rare Anemias, file dfa8b9a7-ccf3-748b-e053-3a05fe0a3a96
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180
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A holistic approach to iron chelation therapy in transfusion-dependent thalassemia patients with serum ferritin below 500 μg/L, file dfa8b9a1-bd95-748b-e053-3a05fe0a3a96
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160
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Curing hemoglobinopathies : challenges and advances of conventional and new gene therapy approaches, file dfa8b9a1-2b36-748b-e053-3a05fe0a3a96
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121
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2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance, file dfa8b9a2-3e39-748b-e053-3a05fe0a3a96
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120
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Finding and treating gaucher disease type 1 - The role of the haematologist, file dfa8b99d-c8e1-748b-e053-3a05fe0a3a96
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117
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Iron deficiency from the standpoint of cardiac rehabilitation : Novel therapeutic opportunities, file dfa8b9a2-aaef-748b-e053-3a05fe0a3a96
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81
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Management of chronic patients during the COVID-19 pandemic: the experience of a referral center for rare hematological disorders in the hardest-hit region in Italy, file dfa8b9a5-334c-748b-e053-3a05fe0a3a96
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78
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Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers, file dfa8b99d-49b7-748b-e053-3a05fe0a3a96
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77
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Finding and treating gaucher disease type 1 - The role of the haematologist, file dfa8b99d-fd84-748b-e053-3a05fe0a3a96
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74
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Advancing the care of β-thalassaemia patients with novel therapies, file dfa8b9a8-453e-748b-e053-3a05fe0a3a96
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69
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Alternative Pathway Involvement in Protoporphyria Patients Related to Sun Exposure, file dfa8b9a5-9982-748b-e053-3a05fe0a3a96
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68
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Acquired Refractory Iron Deficiency Anemia, file dfa8b9a7-ccf6-748b-e053-3a05fe0a3a96
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65
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Rise of the planet of rare anemias: An update on emerging treatment strategies, file c7bdd893-1a4b-4138-b130-fae8666c298d
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63
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Predicting the probability of Gaucher disease in subjects with splenomegaly and thrombocytopenia, file dfa8b9a5-2937-748b-e053-3a05fe0a3a96
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61
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Differential redox state and iron regulation in chronic obstructive pulmonary disease, acute respiratory distress syndrome and coronavirus disease 2019, file dfa8b9a8-7e2d-748b-e053-3a05fe0a3a96
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60
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Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease, file 37b44212-70ad-4e3d-9450-033ca86bb168
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53
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Autoimmune Hemolytic Anemia as a Complication of Congenital Anemias. A Case Series and Review of the Literature, file dfa8b9a7-c961-748b-e053-3a05fe0a3a96
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49
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Thalassaemia is paradoxically associated with a reduced risk of in-hospital complications and mortality in COVID-19: Data from an international registry, file ea54128a-e855-4d4c-ad96-8ead9ccffdc8
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44
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$\upalpha$-Lipoic Acid Improves Hepatic Metabolic Dysfunctions in Acute Intermittent Porphyria: A Proof-of-Concept Study [alpha-Lipoic Acid Improves Hepatic Metabolic Dysfunctions in Acute Intermittent Porphyria: A Proof-of-Concept Study], file dfa8b9a9-a326-748b-e053-3a05fe0a3a96
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43
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Interpreting Iron Homeostasis in Congenital and Acquired Disorders, file 8bf9d137-9c60-4ffc-a71a-7a19d1d877d9
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42
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The α-Lipoic Acid Improves Hepatic Metabolic Dysfunctions in Acute Intermittent Porphyria: A Proof-of-Concept Study, file dfa8b9a9-a324-748b-e053-3a05fe0a3a96
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38
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Migalastat treatment in a kidney-transplanted patient with fabry disease and n215s mutation: The first case report, file dfa8b9a9-ce39-748b-e053-3a05fe0a3a96
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25
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Treating Thalassemia Patients with Luspatercept: An Expert Opinion Based on Current Evidence, file 8e726cc5-705f-4820-a681-ac4d47ca3825
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19
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Usefulness of fibrosis-4 (FIB-4) score and metabolic alterations in the prediction of SARS-CoV-2 severity, file 9fa35cbb-91bd-41a0-8f7b-efbedf47af0c
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18
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Heme Biosynthetic Gene Expression Analysis With dPCR in Erythropoietic Protoporphyria Patients, file b9aeba4d-058c-4617-970c-52684b43df51
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18
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Italian patients with hemoglobinopathies exhibit a 5-fold increase in age-standardized lethality due to SARS-CoV-2 infection, file dfa8b9a9-cec8-748b-e053-3a05fe0a3a96
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16
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Clinical characteristics and outcomes of vaccinated patients hospitalised with SARS-CoV-2 breakthrough infection: Multi-IPV, a multicentre study in Northern Italy, file d62fcb8c-c16a-4513-be3d-a2fb4770728d
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14
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2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance, file dfa8b9a7-9868-748b-e053-3a05fe0a3a96
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9
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Investigational drugs in phase I and phase II clinical trials for thalassemia, file dfa8b99a-347f-748b-e053-3a05fe0a3a96
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8
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Management of age-associated medical complications in patients with β-thalassemia, file dfa8b9a1-0302-748b-e053-3a05fe0a3a96
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8
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Reactivation of developmentally silenced globin genes by forced chromatin looping, file dfa8b991-f2f5-748b-e053-3a05fe0a3a96
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6
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Anemia in Clinical Practice-Definition and Classification : does Hemoglobin Change With Aging?, file dfa8b995-3af8-748b-e053-3a05fe0a3a96
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6
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Treatment with ferric carboxymaltose in stable patients with severe iron deficiency anemia in the emergency department, file dfa8b9a0-34e7-748b-e053-3a05fe0a3a96
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5
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Anemia in elderly hospitalized patients : prevalence and clinical impact, file dfa8b996-79ee-748b-e053-3a05fe0a3a96
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4
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The autophagy-activating kinase ULK1 mediates clearance of free α-globin in β-thalassemia, file dfa8b9a0-4ed9-748b-e053-3a05fe0a3a96
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4
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Usefulness of fibrosis-4 (FIB-4) score and metabolic alterations in the prediction of SARS-CoV-2 severity, file 0f09390e-bc0c-4517-84a5-5e8a4979c83a
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3
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Dyspnoea : focus on the chest but don’t forget the rest, file dfa8b991-c4d6-748b-e053-3a05fe0a3a96
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3
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A giant adrenal myelolipoma in a beta-thalassemia major patient : Does ineffective erythropoiesis play a role?, file dfa8b995-ac69-748b-e053-3a05fe0a3a96
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3
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Clinical Complications and Their Management, file dfa8b99a-6218-748b-e053-3a05fe0a3a96
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3
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Prevalence and predictors of liver fibrosis evaluated by vibration controlled transient elastography in type 1 Gaucher disease, file dfa8b99b-d583-748b-e053-3a05fe0a3a96
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3
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Circulating cell-free DNA and ineffective erythropoiesis in nontransfusion-dependent β-thalassemia, file dfa8b99d-0d10-748b-e053-3a05fe0a3a96
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3
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Redefining thalassemia as a hypercoagulable state, file dfa8b99d-e31b-748b-e053-3a05fe0a3a96
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3
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Longitudinal changes in serum ferritin levels correlate with measures of hepatic stiffness in transfusion-independent patients with beta-thalassemia intermedia, file dfa8b99d-faa0-748b-e053-3a05fe0a3a96
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3
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Care of patients with hemoglobin disorders during the COVID-19 pandemic: An overview of recommendations, file dfa8b9a1-dc86-748b-e053-3a05fe0a3a96
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3
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Beta thalassemia : New therapeutic options beyond transfusion and iron chelation, file dfa8b9a2-8317-748b-e053-3a05fe0a3a96
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3
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Ferric carboxymaltose for sub-acute and chronic iron deficiency anemia in inherited platelet function defects, file dfa8b9a4-382e-748b-e053-3a05fe0a3a96
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3
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Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial, file 7cdc8f3b-17d6-480f-982c-3e1bd756e547
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2
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New therapeutic targets in transfusion-dependent and -independent thalassemia, file dfa8b99a-444b-748b-e053-3a05fe0a3a96
|
2
|
A multicentre observational study for early diagnosis of Gaucher disease in patients with splenomegaly and/or thrombocytopenia, file dfa8b99a-f309-748b-e053-3a05fe0a3a96
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2
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Diagnosis of chronic anaemia in gastrointestinal disorders: A guideline by the Italian Association of Hospital Gastroenterologists and Endoscopists (AIGO) and the Italian Society of Paediatric Gastroenterology Hepatology and Nutrition (SIGENP), file dfa8b99d-f312-748b-e053-3a05fe0a3a96
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2
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Hyperferritinemia and Diagnosis of type 1 Gaucher Disease, file dfa8b9a1-9a03-748b-e053-3a05fe0a3a96
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2
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Effect of Migalastat on cArdiac InvOlvement in FabRry DiseAse: MAIORA study, file eb59dd0a-3570-469e-aded-a65722419d44
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2
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Acute liver injury after SARS-CoV-2 vaccination and luspatercept administration in a patient with β-thalassemia, file 6d6e4867-abb2-4ad1-ae0a-e7ab0397699f
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1
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Similarities and differences between Gaucher disease and acid sphingomyelinase deficiency: An algorithm to support the diagnosis, file 86603425-823d-4c84-accf-246a75e3509d
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1
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Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia, file bc4cfd80-4141-425f-97a1-fb15f0cb4b56
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1
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Deciphering cytopenias in internal medicine: a single-center observational study, file c7f90a23-99e9-4e2e-9542-ea5954952a04
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1
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Advancing the care of β-thalassaemia patients with novel therapies, file d72202d9-1cf0-4de0-a2b9-092524f42f13
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1
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Deferasirox: an orphan drug for chronic iron overload in non-transfusion dependent thalassemia syndromes, file dfa8b99d-d82d-748b-e053-3a05fe0a3a96
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1
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Prognostic role of transferrin saturation in heart failure patients, file dfa8b9a6-3423-748b-e053-3a05fe0a3a96
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1
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Clinical risk scores for the early prediction of severe outocomes in patients hospitalized for {COVID}-19: comment, file dfa8b9a7-e3bb-748b-e053-3a05fe0a3a96
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1
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Clinical factors associated with death in 3044 COVID-19 patients managed in internal medicine wards in Italy : comment, file dfa8b9a8-548d-748b-e053-3a05fe0a3a96
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1
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Epidemiological shift of glucose-6-phosphate dehydrogenase mutations in Northern Italy in the last 15 years, file dfa8b9a8-661f-748b-e053-3a05fe0a3a96
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1
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Splenomegaly: Dare to think rare, file dfa8b9aa-5821-748b-e053-3a05fe0a3a96
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1
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Beta thalassemia : New therapeutic options beyond transfusion and iron chelation, file dfa8b9aa-611a-748b-e053-3a05fe0a3a96
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1
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Totale |
2.992 |