All Genetic disorders articles
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NewsBiogen/Stoke’s zorevunersen hailed as potential first disease-modifying drug for rare epilepsy
Clinical readout for Dravet syndrome trial part of the collaboration the companies signed in 2025.
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NewsUCB wins CHMP favour for first thymidine kinase 2 deficiency treatment
If approved by the European Commission, Kygevvi would provide a new treatment option for eligible patients with the ultra-rare mitochondrial disease in Europe.
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NewsCHMP meeting highlights - May 2025
The EMA’s committee has granted positive opinions for ten new treatments, including medicines for oncology indications and rare diseases.
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NewsNew mass spectrometry Chapter could “revolutionise” biopharma quality assurance
Launch of the USP general method chapter signals a breakthrough in pharmaceutical drug impurity control, according to biopharma experts.
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ArticleEnzyme replacement therapy: improving outcomes in rare disease
In this article, Dr Alaa Hamed, Global Head of Medical Affairs for Rare Disease at Sanofi, shares why the company’s enzyme replacement therapy Xenpozyme® (olipudase alfa) has promise for the rare genetic disease acid sphingomyelinase deficiency (ASMD).
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NewsCHMP meeting highlights - June 2024
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines, including positive opinions for treatments for rare disorders.
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ArticleModifier gene therapy - clinical development and manufacturing considerations
Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the company’s promising modifier gene therapy candidates for ophthalmic disorders.
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NewsNew acquisition to support global gene therapy manufacturing
Acquisition of Forge Biologics by Ajinomoto Co., Inc. is set to extend global capabilities in AAV and plasmid gene therapy manufacturing for Ajinomoto, Co. Inc.
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NewsCHMP meeting highlights – May 2023
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.