News
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TIF STUDY | Patient and Caregiver Perceptions of Thalassaemia Care in 2023
The study revealed that most patients with thalassaemia are receiving adequate treatment, including regular blood transfusions, chelation therapy, proper monitoring, and suitable imaging. Out of 2082 participants, 79.5% began transfusion…
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WHA77 SIDE EVENT | Access to Novel Therapies Globally: Opportunities and Challenges
The Side Event entitled “Access to Novel Therapies Globally: Opportunities and Challenges” is set to take place Friday, 31 May 2024, from 10:00 to 13:30, at the International Museum of…
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BREAKING NEWS | TIF Applauds the EU Parliament’s Adoption of the SoHO Regulation
The new rules modernize the existing directives, now two decades old, and are aimed at addressing challenges and weaknesses identified through the years, including, inter alia, the risk of shortages,…
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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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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 to reduce the sickling and disintegration of red blood cells…
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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, and share concerns and challenges. A unique occasion for a…
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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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