1,572 results on '"Cappellini,Maria Domenica"'
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2. Acute liver injury after SARS-CoV-2 vaccination and luspatercept administration in a patient with β-thalassemia
3. Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease
4. Acid sphingomyelinase deficiency (ASMD): addressing knowledge gaps in unmet needs and patient journey in Italy—a Delphi consensus
5. Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial
6. RNA polymerase II pausing temporally coordinates cell cycle progression and erythroid differentiation
7. Anemia management and transfusion strategy in internal medicine units: Less is more
8. Magnitude of cumulative iron overload correlates with the severity of anemia in untreated non-transfusion-dependent β-thalassemia
9. Similarities and differences between Gaucher disease and acid sphingomyelinase deficiency: An algorithm to support the diagnosis
10. Disorders of Iron Metabolism
11. Long-term bone outcomes in Italian patients with Gaucher disease type 1 or type 3 treated with imiglucerase: A sub-study from the International Collaborative Gaucher Group (ICGG) Gaucher Registry
12. Step out of the shadows: a call for action for rare diseases
13. Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial
14. Iron metabolism and iron deficiency anemia in women
15. Mortality in β-thalassemia patients with confirmed pulmonary arterial hypertension on right heart catheterization
16. Decrease in Hemoglobin Levels during Acute Attacks in Patients with Idiopathic Recurrent Pericarditis: A Model of Anemia in Acute Disease.
17. Anemia and iron overload as prognostic markers of outcomes in β-thalassemia.
18. Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
19. Tailored diagnostic decision tree resulting from machine learning to improve early diagnosis of ASMD
20. Transfusion Independence after Exagamglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia
21. Impact of implementing a Choosing Wisely educational intervention into clinical practice: The CW-SIMI study (a multicenter-controlled study)
22. The use of luspatercept for thalassemia in adults
23. Recommendations for diagnosis, treatment, and prevention of iron deficiency and iron deficiency anemia.
24. Fyn specifically Regulates the activity of red cell glucose-6-phosphate-dehydrogenase
25. Liver involvement in Gaucher disease: A practical review for the hepatologist and the gastroenterologist
26. Variations in hemoglobin level and morbidity burden in non-transfusion-dependent β-thalassemia
27. Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation
28. Treatment with ferric carboxymaltose in stable patients with severe iron deficiency anemia in the emergency department
29. Ferric carboxymaltose for sub-acute and chronic iron deficiency anemia in inherited platelet function defects
30. Hospital Care of Older Patients With COPD: Adherence to International Guidelines for Use of Inhaled Bronchodilators and Corticosteroids
31. Oocyte quality in women with thalassaemia major: insights from IVF cycles
32. 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)
33. Morbidity-free survival and hemoglobin level in non-transfusion-dependent β-thalassemia: a 10-year cohort study
34. How I manage medical complications of β-thalassemia in adults
35. A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?
36. Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent
37. Thalassaemia
38. Risk factors for heart disease in transfusion-dependent thalassemia: serum ferritin revisited
39. Common fetal hemoglobin variants in Lebanese patients bearing the codon 29 beta gene mutation associated with different thalassemia phenotypes
40. Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia
41. An unusual diagnosis in a 31-year-old man with abdominal pain and hyponatremia
42. Profile of Luspatercept in the Treatment of Anemia in Adults with Non-Transfusion-Dependent β-Thalassemia (NTDT): Design, Development and Potential Place in Therapy
43. Disorders of Hemoglobin Synthesis: Pathophysiology and Diagnostic Evaluation
44. Clinical management of iron deficiency anemia in adults: Systemic review on advances in diagnosis and treatment
45. Parkinson’s disease in Gaucher disease patients: what’s changing in the counseling and management of patients and their relatives?
46. 148 - The Thalassemias
47. What is behind a relapse of thrombotic thrombocytopenic purpura?
48. Low-dose Synachten test with measurement of salivary cortisol in adult patients with β-thalassemia major
49. Health‐related quality of life in patients with β‐thalassemia: Data from the phase 3 BELIEVE trial of luspatercept
50. Management of luspatercept therapy in patients with transfusion‐dependent β‐thalassaemia
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