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EAHAD 2026: Nine-Year Outcomes of Gene Therapy
February 2026
Nine-year data show sustained safety and reduced bleeding after gene therapy in haemophilia B, although enrichment for full viral capsids did not deliver clear clinical advantages.
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9
Mins
28 Jul 2016
Thrombocytopenia Caused By Inherited Haematopoietic Transcription Factor Mutation: Clinical Phenotypes and Diagnostic Considerations
Inherited thrombocytopenias comprise a heterogeneous group of blood disorders with abnormalities in genes related to glycoproteins and adhesion molecules…
2
Mins
28 Jul 2016
The Spectrum of Late Effects After Cancer: Lessons from Paediatric Oncology and the Value of International Collaboration
This presentation described the occurrence of late side effects (commonly described as ‘late effects’) in childhood cancer survivors (CCS), and how…
1
Mins
28 Jul 2016
Chronic Lymhocytic Leukaemia: Treatment of the Elderly
Chronic lymphocytic leukaemia (CLL) mainly affects elderly patients.1 With the increasing comorbidity burden and frailty in elderly patients, evaluation…
9
Mins
28 Jul 2016
The Relative Contributions of Germline Variation, Epimutation, and Somatic Mutation to Paediatric Leukaemia Predisposition
The next-generation sequencing era has repeatedly demonstrated that the amount of acquired somatic mutations in paediatric cancers can rarely account for…
16
Mins
28 Jul 2016
Current Management of Adult Acute Lymphoblastic Leukaemia: Emerging Insights and Outstanding Questions
Less than 50% of patients with adult acute lymphoblastic leukaemia (ALL) experience long-term survival and for those adults >60 years old, long-term…
7
Mins
28 Jul 2016
Management of Children With Sickle Cell Disease in Europe: Current Situation and Future Perspectives
Sickle cell disease (SCD) is the most common haemoglobinopathy worldwide and its frequency has steadily increased in Europe in the past decades. SCD is a…
12 Jul 2016
Introducing EMJ Neurology 4.1
Welcome to EMJ Neurology 4.1, an edition filled with informative and engaging content which spotlights the latest research and developments in the field…
3
Mins
12 Jul 2016
A Call from a Patient to Openly Discuss Body Image in the Treatment of Type 1 Diabetes
Diabetes is a non-communicable disease (NCD) meaning that it often cannot be seen. After 20 years of living with Type 1 diabetes I am lucky to have no…
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