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Sarepta dives deeper into gene therapy
https://pharmaphorum.com/news/sarepta-dives-deeper-gene-therapy/
Sarepta
Pharma collaborates to improve chances of NICE backing Duchenne drugs
Project will initially focus on DMD but could benefit other rare diseases
FDA reviewers slam PTC’s Duchenne drug ahead of meeting
FDA staff not persuaded by patchy clinical trial evidence for Translarna.
EU regulators reject Santhera’s Raxone for DMD
Swiss company to appeal regulators’ decision.
UK patients gain early access to Duchenne drug
Patients to get Santhera’s Raxone under EAMS scheme.
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