Two rare disease therapies approved in Scotland
Drug Discovery World
JULY 9, 2024
The Scottish Medicines Consortium (SMC) has published final advice on two rare disease therapies. Elfabrio (pegunigalsidase alfa), a novel enzyme replacement therapy (ERT), has been approved as a treatment option for Fabry disease in adults. Also, birch bark extract gel has been approved as a treatment option for junctional and dystrophic epidermolysis bullosa (EB), subject to data collection under the SMC’s ultra-orphan medicines framework.
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