News
-
Oxbryta Shows Promise in Reducing Cell Sickling in Pediatric SCD Patients
In a promising leap forward for pediatric sickle cell disease (SCD) treatment, the oral medication Oxbryta (voxelotor) has been shown to reduce the sickling and disintegration of red blood cells…
Read More » -
EVENT | 1st RAIN Summit for Patients with Rare Anaemias | 18-19 May 2024
The workshop will present an excellent opportunity for patients to exchange knowledge on the latest scientific advances for Rare Anaemias, and share concerns and challenges. A unique occasion for a…
Read More » -
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…
Read More » -
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…
Read More » -
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…
Read More » -
-
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…
Read More » -
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…
Read More » -
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…
Read More » -
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…
Read More »