Recommendation from NICE for PTC Therapeutics gene therapy
Eladocagene exuparvovec is for children with ultra-rare genetic disorder in final draft guidance
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Tag: PTC Therapeutics
NICE recommends PTC Therapeutics’ Translarna reimbursement
The NICE recommendation followed data from clinical trials and real-world evidence
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PTC Therapeutic’s Upstaza has been recommended by both the EMA and CHMP
It is the first gene therapy to be directly administered into the brain to treat aromatic L-amino acid decarboxylase (AADC) deficiency
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