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NICE recommends gene therapy for inherited retinal dystrophy
BMJ 2019; 366 doi: https://doi.org/10.1136/bmj.l5394 (Published 04 September 2019) Cite this as: BMJ 2019;366:l5394
- Elisabeth Mahase
- The BMJ
Draft guidance from the National Institute for Health and Care Excellence (NICE) has recommended licensed gene therapy for people with vision loss caused by RPE65 mediated inherited retinal dystrophy who have enough viable retinal cells, after a deal between NICE and the manufacturer Novartis.1
RPE65 mediated inherited retinal dystrophies are rare and involve progressive vision loss that leads to near total blindness. So far treatment has been limited to supportive care.
NICE’s review of clinical trial data …
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