Catherine Skari

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…

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The Italian Breakthrough in CRISPR trials for Rare Diseases: A focus on β-Thalassemia and Sickle Cell Disease Treatment

The development of gene therapy and the current advantageous method of clustered regularly interspaced short palindromic repeats (CRISPRs) has allowed…

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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…

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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…

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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…

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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…

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BREAKING NEWS | TIF’s Collaboration with WHO Extended to 2027

Our enduring collaboration was newly endorsed by WHO’s Executive Board at its 154th session in January 2024, demonstrating our significant…

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APLASTIC ANEMIA | Incidence and Clinical Management in Spain Consistent with Other Studies

The study was carried out through a multicenter, ambispective, observational approach encompassing 7 Spanish hospitals, catering to roughly 3.91 million…

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ADVOCACY IN ACTION | Highlights from the TIF Meeting with Greek Health Minister, Adonis Georgiadis

A productive meeting between the Greek Minister of Health, Mr. Adonis Georgiades, and a Thalassaemia International Federation (TIF) delegation took…

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TIF INTERNATIONAL CONFERENCE | 3-5 November 2023

Inside the Conference The 16th International Conference on Thalassaemia and Other Haemoglobinopathies, jointly organised with the 18th TIF Conference for…

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