Catherine Skari
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Apr- 2024 -16 April
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15 AprilScientific Literature
Defining Curative Endpoints for Transfusion-Dependent β-Thalassemia in the Era of Gene Therapy and Gene Editing
β-thalassemia is a monogenic disease that results in varying degrees of anemia. In the most severe form, known as transfusion-dependent…
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11 April
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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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