Rare Diseases

In proud collaboration with EBMT, we have captured the highlights from the recent 52nd Annual Meeting, EBMT 2026. Below, hear from Dr Anna Sureda, President of EBMT, Dr Rafael Duarte, Congress President, and other world-leading experts sharing some of the most ...

At EBMT26, Prof Claire Booth presented a plenary session on gene therapy in primary immunodeficiencies. In this interview, she reflects on the field’s most important milestones, how gene therapy compares with allogeneic transplantation, and the critical challenge of ensuring patients can actually access these transformative treatments.

Three experts discuss the latest advances in PNH pathophysiology, diagnosis and therapeutics.

As part of our Haematology Horizons practice pearl series, Dr Douglas Tremblay (Icahn School of Medicine at Mount Sinai, New York City, NY, USA) explores the contemporary role and limitations of HMAs across chronic myelomonocytic leukaemia subtypes, contrasts treatment goals by patient fitness and transplant eligibility and looks ahead to emerging HMA combinations and novel agents designed specifically for CMML.

Watch expert haematologists discuss managing cytopenias in patients with myelofibrosis.

The 67th ASH Annual Meeting and Exposition spotlighted many pivotal trial results across non-malignant haematology including immune thrombocytopenia, bleeding disorders, iron deficiency, von Willebrand disease, cancer-associated hematologic complications and more. We break down some of the most important abstracts presented, and what this might mean for future clinical practice.

A leading sickle cell disease expert discusses burden, treatment and strategies for effective lifespan care.

INCA33989, a mutant CALR-specific antibody, showed an 80% platelet normalization rate and no dose-limiting toxicities in early-phase trials for essential thrombocythemia. Dr. John Mascarenas highlights the agent’s potential as a disease-modifying therapy, with reductions in mutant CALR burden and strong tolerability—marking a significant step forward in ET treatment.

Physician burnout is at a critical point. In this episode, Nicky speaks with Dr Alfred Atanda about why so many physicians are burning out and what can be done to change the trend. From personal experience to system-wide solutions, Dr Atanda shares valuable insights on improving physician well-being and building a more effective healthcare culture.