news
NICE recommends new Fabry disease treatment
Recommendation of Elfabrio® (pegunigalsidase alfa) means patients with Fabry disease in England will be able to access a new treatment option.
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Fabry disease
news
Enzyme replacement therapy approved for Fabry disease
The European Commission (EC) has given approval to the first PEGylated enzyme replacement therapy to treat Fabry disease.
news
EMA’s human medicines committee (CHMP) meeting highlights – February 2023
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended eight new medicines for approval, including an enzyme replacement therapy for a rare disease.
news
CHMP 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.
news
EC grants Orphan Drug Designation to FLT190, a Fabry Disease treatment
FLT190 gene therapy has been granted Orphan Drug Designation based on preliminary trial data and the positive opinion of an EMA committee.
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Capsosomes: the revolutionary enzyme carriers
Nature can offer inspiration for the synthesis of more effective drug delivery platforms. In this article, Marc Baiget Francesch discusses the potential of capsosomes as a novel, biocompatible, drug delivery system.
