EU
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News
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 the FANCA gene. Mutations in FANCA hinder DNA repair and occur…
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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…
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TIF News
NEW WEBINAR | Empowering Patient Leaders: Understanding EU Health Policies
📌 Webinar Title: ‘‘Empowering Patient Leaders: Understanding EU Health Policies’’ 📌 Date & Time: 18 December 2023, Monday, at 14:00 EET 📌 Registration: https://forms.office.com/e/nTz4yTd9NA In this webinar, we aim to…
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TIF News
TIF’s NEW e-COURSE | Enhance Your Understanding of EU Health Policy
To address this need, the Thalassaemia International Federation (TIF) has launched an e-course titled ‘EU Health Policy Course’, which offers a comprehensive and up-to-the-minute overview of EU health-related policies. The…
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POSITION PAPER | Strengthening the Resilience of EU Health Systems
Thalassaemia is more than just a rare condition in Europe; it is a pressing issue that requires prompt action. With shifting migration patterns and escalating healthcare costs, EU health systems…
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News
TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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NEW APPROVAL | PYRUKYND® Gets EU Green Light For Pyruvate Kinase (PK) Deficiency
The European Commission has granted, on November 10, 2022, marketing authorization to Agios Pharmaceuticals for PYRUKYND® intended for the treatment of PK deficiency in adult patients. People with PK…
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News
POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells
This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal…
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TIF News
THALIA2018-2021 | Europe Is Seeing A Surge In Thalassaemia, Project Results Show
“Although thalassemia has always been endemic in Europe, especially in southern European countries such as Cyprus, Greece and Italy, the disease now knows no borders and constitutes an increasing health,…
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