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MHRA approves first gene therapy for infusion into the brain
Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.
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PTC Therapeutics
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Roche awarded 2022 Drug Discovery of the Year for Evrysdi®
The development team behind Roche’s Evrysdi® (risdiplam), the first mRNA splicing modifier to be approved, have been honoured with the BPS’s Drug Discovery of the Year Award 2022.
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EMA accepts marketing application for Spinal Muscular Atrophy treatment
The European Medicines Agency (EMA) has accepted the marketing authorisation application for Evrysdi™ (risdiplam) as a treatment of spinal muscular atrophy (SMA).
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NICE allows extra time for agreement on ataluren price
4 May 2016 | By Victoria White, Digital Content Producer
NICE has allowed extra time for further discussions on access arrangements for PTC Therapeutics’ Translarna (ataluren) for Duchenne Muscular Dystrophy...
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PTC submits EU application for Translarna for cystic fibrosis
30 September 2015 | By Victoria White
If approved, Translarna would be the first oral protein restoration treatment that targets the underlying cause of nonsense mutation cystic fibrosis...
