All Rare diseases articles – Page 3
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VideomRNA therapies: a new frontier
Nicolas Chornet, Senior Vice President of International Manufacturing at Moderna, speaks to EPR about the future of mRNA therapies and offers an update on the company’s international expansion plans.
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NewsMSD medicine accepted for use in Scotland
Scotland is the first European country to accept the HIF-2α inhibitor WELIREG® (belzutifan) for eligible adults with von Hippel-Lindau (VHL) disease.
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ArticleExploring the gene therapy biomanufacturing landscape
In this exclusive Q&A with European Pharmaceutical Review, Sheila Ann Mikhail, co-founder of AskBio and keynote speaker at CPHI Barcelona delves into the current landscape and future potential of the gene therapy sector. She explores strategies for making gene therapies more affordable and accessible, innovations in drug delivery, as ...
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NewsSmall molecule drug shows promise in rare disease
In a Phase II trial, the small-molecule drug CBL-514 demonstrated significant reduction in lipoma size and pain improvement in Dercum's disease, a rare disorder.
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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.
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News$1.7b acquisition to develop rare haematology therapy
When CTI BioPharma is acquired by Swedish Orphan Biovitrum AB (Sobi), the companies intend to advance an oral kinase inhibitor treatment for a rare haematological disease.
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NewsTakeda to build £600m plasma therapy manufacturing site
Takeda’s largest manufacturing capacity expansion investment in Japan will fund the build of a new facility for plasma-derived therapies.
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NewsCHMP recommends first pegylated enzyme for Fabry disease
A PEGylated enzyme replacement therapy for Fabry disease has received recommendation for a marketing authorisation from the European Medicines Agency (EMA)'s human medicines committee.
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