News
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REGULATORY NEWS | EU Backs Revolutionary Oral Monotherapy for Adult PNH Patients
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use…
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Recent Study Links Transfusion-Dependent Thalassaemia to Adverse Maternal, Neonatal Outcomes
A meta-analysis of 15 studies found that pregnant women with β-thalassaemia major and β-thalassaemia intermedia were at greater risk of heart failure, venous thromboembolism, death, and other adverse neonatal and…
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INTERNATIONAL THALASSAEMIA DAY 2024 | The Official Campaign is Live!
The Theme May 8, 2024, is International Thalassaemia Day. The theme of the event this year is: “Empowering Lives, Embracing Progress: Equitable and Accessible Thalassaemia Treatment for All”. Our…
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EXCLUSIVE WEBINAR | Guidelines for the Management of a-Thalassaemia |14 March 2024
📌 Webinar Title: Guidelines for the Management of α-Thalassaemia 📌 Date & Time: Thursday, March 14, 2024, 13:00 Bangkok / 07:00 Paris / 22:00 PST This session aims to…
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SPOTLIGHT | RAIN Endorses the First International Guidelines for Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is the most common cause of chronic congenital non-spherocytic haemolytic anaemia, impacting between one in 100,000 to one in 300,000 individuals globally. Those with PKD suffer…
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REGULATORY NEWS | AstraZeneca’s Voydeya Recommended for PNH Treatment in EU
Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who…
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Pain Impact and Management in Individuals With Transfusion-Dependent Thalassaemia
Over the last several decades, there has been significant progress in addressing the main symptoms and related health issues of people with transfusion-dependent thalassemia (TDT). Yet, the problem of pain…
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ELEVATING AWARENESS | 29 February Is Rare Disease Day
Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.…
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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Ruxoprubart Scores FDA Orphan Drug Designation for PNH Treatment
FDA’s recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition. Ruxoprubart is currently undergoing a…
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