All Rare diseases articles – Page 2
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NewsAAV gene therapy manufacturing gets £1.4m boost
The funding aims to reduce the costs of AAV production for gene therapies, helping to accelerate treatment access for patients with rare diseases.
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ArticleRegulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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NewsPfizer receives EU haemophilia B gene therapy approval
The single-dose gene therapy was approved for certain patients with haemophilia B in the US and Canada earlier this year, marketed as BEQVEZ.
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ArticleDrug approval roundup – May/June 2024
This article highlights some of the key regulatory approvals granted in May and June in the EU and US so far this year, including for oligonucleotide and biologic therapies.
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NewsFive-year data from Genentech SMA study released
Spinal muscular atrophy (SMA) patients in the long-term extension Evrysdi study maintained or improved key developmental skills over the five-year period, data shows.
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ArticleWhy patient-centricity is essential to accelerate rare disease clinical research
There is an urgent need for novel treatment options for people living with rare diseases, but carrying out clinical research in this field is notoriously difficult. As Clinical Trials Day approaches, Cezary Statuch, Head of Medical Affairs International and Japan at Alexion Pharmaceuticals, highlights how adopting patient-centric approaches can help ...
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