News
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TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia
The Thalassaemia International Federation (TIF) expresses its disappointment about the outcome of the reimbursement negotiation process concerning gene therapy (Zynteglo™) for transfusion-dependent thalassaemia patients, announced last week. The Federation’s distress…
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RARE DISEASE DAY 2022 | Raising Awareness For People With Rare Diseases
Rare Disease Day is the movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease everywhere…
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PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
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Bluebird puts €1.58m price on gene therapy Zynteglo
Bluebird bio Inc. set a price for its gene therapy, Zynteglo, at 1.58 million euros ($1.78 million) over five years, after winning conditional approval in Europe earlier this month…
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