News
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BREAKING | Vertex and CRISPR’s Gene-editing Therapy Casgevy Receives Early FDA Approval for β-Thalassaemia Treatment
Just one month prior, Casgevy made history as the first CRISPR gene editing therapy to enter the US market when it was approved for sickle cell disease. Now, the FDA…
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EXCLUSIVE WEBINAR | Guidelines for the Management of Non-Transfusion Dependent β-Thalassaemia
📌 Webinar Title: Guidelines for the Management of Non-Transfusion Dependent β-Thalassaemia 📌 Date & Time: Friday, February 02, 2024, 15:00 CET Recognizing the Clinical Relevance of NTDT Non‐transfusion‐dependent thalassaemias…
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Chinese Base Editing Therapy ‘Clinically Cured’ Patient with β-Thalassaemia, Researchers Say
Shanghai-based biotech company CorrectSequence Therapeutics, in collaboration with Guangxi Medical University, has achieved a significant clinical milestone by successfully treating a young patient with transfusion-dependent β-thalassaemia using their base editing…
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COLD AGGLUTININ DISEASE | ANX1502 Passes Phase 1 Trial, Proving Safe for Healthy Volunteers
Administered orally as a liquid suspension, ANX1502 demonstrated good tolerability, with no serious side effects reported. The drug’s pharmacokinetics also supported twice-daily dosing for a future proof-of-concept study. “We are…
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A YEAR IN RETROSPECT | Global Public Health Achievements and Challenges in 2023
We applaud the remarkable strides in eradicating diseases and infections, such as ending the COVID-19 public health emergency and intensifying efforts to eliminate polio. Additionally, we celebrate the progress in…
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wAIHA | Fostamatinib May Induce Durable Hemoglobin Response in Subset of Patients
Interestingly, this positive outcome was predominantly observed among participants hailing from North America, Australia, or Western Europe, while those from Eastern Europe didn’t show the same results. The current treatment…
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SICKLE CELL DISEASE | FDA Approves Two Landmark Gene Therapies for the Treatment of the Disorder
The two therapies are Casgevy (exa-cel) by Vertex Pharmaceuticals and CRISPR Therapeutics – the world’s first drug to utilize the revolutionary CRISPR gene-editing system which earned its creators the 2020…
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NEWS | ICER Releases Draft Evidence Report on Treatment for Paroxysmal Nocturnal Hemoglobinuria
This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions. Register…
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PHARMA NEWS | FDA Approves Iptacopan for Paroxysmal Nocturnal Hemoglobinuria
Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside…
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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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