PTC Therapeutics Inc at Jefferies Gene Therapy Summit Transcript
Thanks, everyone, for attending the Jefferies Gene Therapy Summit this afternoon. I'd like to welcome our next company. We have PTC Therapeutics. And I'd like to introduce Jodi Cook, nearest to me, Head of the Gene Therapy and Strategy group, and formerly from Agilis. And then Nikolai Naryshkin, the Vice President of External Research and Alliance Management.
So given this is a gene therapy summit, Jodi, maybe we can start with the Company's AADC deficiency gene therapy program. Can you just maybe go over the clinical data to date? I think you presented and reported on data up to 26 patients. So maybe you can provide that for the audience, and then we'll go into some of the details.
Sure. Thank you very much for having us. Let me tell you a little bit about our AADC program. It's -- AADC is a rare genetic disease where patients are born with the inability actually to make dopamine, so they really have no volitional -- they don't develop volitional motor movement.
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