Catherine Skari
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Apr- 2024 -10 AprilScientific Literature
Systematic Review and Evidence Gap Assessment of the Clinical, Quality of Life, and Economic Burden of Alpha-Thalassemia
A recent evidence gaps assessment of the clinical, health-related quality of life, and economic burden associated with α-thalassemia is lacking.…
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10 April
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6 AprilNews
REGULATORY NEWS | EMA Accepts Rocket Pharma’s Gene Therapy Application for Fanconi Anemia
RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of…
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4 April
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Mar- 2024 -31 MarchNews
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…
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30 MarchNews
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,…
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27 MarchNews
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…
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26 MarchNews
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…
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25 March
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22 MarchNews
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,…
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