| scholarly article | Q13442814 |
| P356 | DOI | 10.1517/21678707.2016.1135793 |
| P50 | author | Alessia Finotti | Q54585393 |
| Nicoletta Bianchi | Q40649466 | ||
| Ilaria Lampronti | Q42427097 | ||
| Cristina Zuccato | Q45888508 | ||
| P2093 | author name string | Roberto Gambari | |
| Monica Borgatti | |||
| P2860 | cites work | European regulation on orphan medicinal products: 10 years of experience and future perspectives. | Q51711644 |
| Noninvasive assessment of liver fibrosis in thalassaemia major patients by transient elastography (TE) â lack of interference by iron deposition | Q53100852 | ||
| Everolimus is a potent inducer of erythroid differentiation and gamma-globin gene expression in human erythroid cells. | Q54568641 | ||
| Effects of rapamycin on accumulation of alpha-, beta- and gamma-globin mRNAs in erythroid precursor cells from beta-thalassaemia patients. | Q54585312 | ||
| cMYB is involved in the regulation of fetal hemoglobin production in adults. | Q54591078 | ||
| Quantification of HBG mRNA in primary erythroid cultures: prediction of the response to hydroxyurea in sickle cell and beta-thalassemia. | Q54791501 | ||
| Beta-thalassemia | Q21202886 | ||
| Oral deferiprone for iron chelation in people with thalassaemia | Q24200309 | ||
| Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin | Q24293797 | ||
| Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia | Q24618997 | ||
| MicroRNA-15a and -16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13 | Q24633081 | ||
| Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia | Q26824116 | ||
| The current status of orphan drug development in Europe and the US | Q26830502 | ||
| Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients | Q28730903 | ||
| Deferasirox | Q29393588 | ||
| Drug repositioning: identifying and developing new uses for existing drugs | Q29614779 | ||
| Growth and puberty and its management in thalassaemia | Q30737438 | ||
| Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel | Q33558770 | ||
| Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy | Q33603146 | ||
| MicroRNA-96 directly inhibits γ-globin expression in human erythropoiesis | Q33987959 | ||
| The US Orphan Drug Act: rare disease research stimulator or commercial opportunity? | Q34090111 | ||
| Pricing and reimbursement of orphan drugs: the need for more transparency | Q34193502 | ||
| Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. | Q34205528 | ||
| Orphan drug development: an economically viable strategy for biopharma R&D. | Q34256983 | ||
| Orphan drug: Development trends and strategies | Q34373817 | ||
| Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease | Q34449867 | ||
| Deferasirox, deferiprone and desferrioxamine treatment in thalassemia major patients: cardiac iron and function comparison determined by quantitative magnetic resonance imaging. | Q34451519 | ||
| Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis | Q34476161 | ||
| New uses for old drugs | Q34661227 | ||
| Two decades of orphan product development | Q34932215 | ||
| Nuclease-mediated gene editing by homologous recombination of the human globin locus | Q35025959 | ||
| Higher doses of lenalidomide are associated with unacceptable toxicity including life-threatening tumor flare in patients with chronic lymphocytic leukemia | Q35033514 | ||
| An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia | Q35127943 | ||
| A pilot study of subcutaneous decitabine in β-thalassemia intermedia | Q35212344 | ||
| A genetic score for the prediction of beta-thalassemia severity. | Q35236041 | ||
| Variants in genetic modifiers of β-thalassemia can help to predict the major or intermedia type of the disease | Q35528718 | ||
| Fertility and pregnancy in thalassemia major | Q36337062 | ||
| Pathophysiology and Clinical Manifestations of the β-Thalassemias | Q36526536 | ||
| Combined chelation therapy in thalassemia major for the treatment of severe myocardial siderosis with left ventricular dysfunction. | Q36532618 | ||
| Alpha and beta cell evaluation in patients with thalassaemia intermedia and iron overload | Q36706761 | ||
| Medicinal chemistry of fetal hemoglobin inducers for treatment of beta-thalassemia | Q36724048 | ||
| Mi2β-mediated silencing of the fetal γ-globin gene in adult erythroid cells. | Q36797277 | ||
| Management of the thalassemias | Q36870253 | ||
| On the road to gene therapy for beta-thalassemia and sickle cell anemia | Q37071706 | ||
| Incentives for orphan drug research and development in the United States | Q37074235 | ||
| Comparison of in-vitro and in-vivo response to fetal hemoglobin production and γ-mRNA expression by hydroxyurea in Hemoglobinopathies | Q37136101 | ||
| Why rare diseases are an important medical and social issue | Q37191857 | ||
| A feedback loop consisting of microRNA 23a/27a and the β-like globin suppressors KLF3 and SP1 regulates globin gene expression | Q37264387 | ||
| The efficacy of iron chelator regimes in reducing cardiac and hepatic iron in patients with thalassaemia major: a clinical observational study | Q37268421 | ||
| CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity | Q37271186 | ||
| Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells | Q37348943 | ||
| Discovering the genetics underlying foetal haemoglobin production in adults. | Q37432250 | ||
| Evaluation of safety and pharmacokinetics of sodium 2,2 dimethylbutyrate, a novel short chain fatty acid derivative, in a phase 1, double-blind, placebo-controlled, single-dose, and repeat-dose studies in healthy volunteers. | Q37524864 | ||
| A phase 2 study of HQK-1001, an oral fetal haemoglobin inducer, in β-thalassaemia intermedia. | Q37524883 | ||
| Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives | Q37581320 | ||
| Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation. | Q37623526 | ||
| Modulators of erythropoiesis: emerging therapies for hemoglobinopathies and disorders of red cell production. | Q37673351 | ||
| Update on thalassemia: clinical care and complications | Q37683933 | ||
| Global burden, distribution and prevention of β-thalassemias and hemoglobin E disorders | Q37810074 | ||
| Gene therapy in thalassemia and hemoglobinopathies. | Q37854468 | ||
| Pricing for orphan drugs: will the market bear what society cannot? | Q48159215 | ||
| Costs and cost-effectiveness of allogeneic stem cell transplantation in children are predictable | Q48457773 | ||
| Efficacy of hydroxyurea in providing transfusion independence in β-thalassemia. | Q50535662 | ||
| Drug repositioning for orphan diseases | Q37866927 | ||
| MicroRNA therapeutics. | Q37869804 | ||
| Complications of thalassemia major and their treatment | Q37888721 | ||
| Targeted therapeutic strategies for fetal hemoglobin induction | Q37967326 | ||
| Alternative options for DNA-based experimental therapy of β-thalassemia. | Q37993118 | ||
| Evidence for rapamycin toxicity in pancreatic β-cells and a review of the underlying molecular mechanisms. | Q38123696 | ||
| Financing drug discovery for orphan diseases | Q38165615 | ||
| Evidence-based focused review of the status of hematopoietic stem cell transplantation as treatment of sickle cell disease and thalassemia | Q38186701 | ||
| Red blood cell transfusions for thalassemia: results of a survey assessing current practice and proposal of evidence-based guidelines | Q38194595 | ||
| Recent trends for novel options in experimental biological therapy of β-thalassemia | Q38220695 | ||
| A systematic review and meta-analysis of deferiprone monotherapy and in combination with deferoxamine for reduction of iron overload in chronically transfused patients with β-thalassemia | Q38259421 | ||
| Establishing medical plausibility in the context of orphan medicines designation in the European Union. | Q38283124 | ||
| Recent trends in the gene therapy of β-thalassemia | Q38367473 | ||
| Priority review drugs approved by the FDA and the EMA: time for international regulatory harmonization of pharmaceuticals? | Q38503395 | ||
| Access to orphan drugs in Europe: current and future issues | Q39659839 | ||
| Thalassaemia | Q39708856 | ||
| Compounds of the anthracycline family of antibiotics elevate human gamma-globin expression both in erythroid cultures and in a transgenic mouse model. | Q39774355 | ||
| Efficacy of Rapamycin as Inducer of Hb F in Primary Erythroid Cultures from Sickle Cell Disease and β-Thalassemia Patients | Q40897603 | ||
| Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine β-thalassemia | Q40915310 | ||
| Use of biomarkers in the context of orphan medicines designation in the European Union | Q41901528 | ||
| An analysis of FDA-approved drugs for metabolic diseases | Q42177399 | ||
| A phase 2 trial of HQK-1001 in HbE-β thalassemia demonstrates HbF induction and reduced anemia | Q42401596 | ||
| Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells | Q42765881 | ||
| Transcriptional silencing of fetal hemoglobin by BCL11A. | Q42936407 | ||
| Ability of deferasirox to bind iron during measurement of iron | Q43156449 | ||
| Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient | Q44451436 | ||
| Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone | Q44451892 | ||
| A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease | Q45377608 | ||
| Gene therapy: Targeting β-thalassaemia | Q45863576 | ||
| Bone marrow as a source of hematopoietic stem cells for human gene therapy of β-thalassemia | Q45864447 | ||
| Genetic therapy for beta-thalassemia: from the bench to the bedside | Q45866403 | ||
| A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia | Q45889126 | ||
| Response to hydroxyurea in beta thalassemia major and intermedia: experience in western India. | Q45958661 | ||
| Thalidomide therapy in a patient with thalassemia major. | Q46624335 | ||
| Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major | Q46864846 | ||
| First human studies with a high-molecular-weight iron chelator | Q47249074 | ||
| Treatment of two infants with Cooley's anemia with sodium phenylbutyrate. | Q47824256 | ||
| P433 | issue | 3 | |
| P921 | main subject | orphan drug | Q1367466 |
| P304 | page(s) | 299-315 | |
| P577 | publication date | 2016-01-22 | |
| P1433 | published in | Expert opinion on orphan drugs | Q27725467 |
| P1476 | title | Orphan Drugs and Potential Novel Approaches for Therapies of β-Thalassemia: Current Status and Future Expectations | |
| P478 | volume | 4 |
| Q47396521 | BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression | cites work | P2860 |
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