Patients
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NOVITÀ
EHA2022 | Agios Presents New Data Supporting the Benefits of PYRUKYND®In Adults With PKD
Data were presented at the European Hematology Association (EHA) Annual Congress, hosted virtually and in person in Vienna on June 9-12, 2022. “Following the FDA approval of PYRUKYND® for the treatment…
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NEW EVENT | Register Now For The 1st PIBA Members’ Meeting!
Watch the Full PIBA Meeting Video Recording HERE.
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INTERNATIONAL THALASSAEMIA DAY | 8 May 2022
Every single one of us has the ability to make a difference, large or small, and together we can make real progress in raising valuable awareness about thalassaemia and its…
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Patients’ stories
IRON BOY: SURVIVING β-THALASSAEMIA MAJOR by Arthur Bozikas
Where was my wife? I found myself sitting alone in front of the obstetrician, who my wife, Helen, and I had met for the first time only minutes earlier. But…
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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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SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic…
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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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TIF Article: COVID-19 Pandemic Exacerbated Existing Problems With Thalassaemia Care
The article discusses how the COVID-19 pandemic has magnified challenges in thalassaemia care worldwide and underscores TIF’s activities throughout the coronavirus outbreak in support of patients with haemoglobinopathies. Hematology Advisor…
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NOVITÀ
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…
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