At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where mHTT was lowered by 21% and 43% respectively, for 5mg and 10mg doses. In addition PTC518 treatment resulted in a notable slowing in progression of motor symptoms at month 12, as assessed by the Total Motor Score (2.0 points worsening for 5mg and 1.3 points worsening for 10mg vs. 4.9 points worsening for placebo).

IHA President Svein Olaf Olsen says “This is really exciting news!  It’s great to see results of this magnitude, and more so because PTC518 is an easy-to-administer oral medication with favourable tolerability.  Congratulations and thanks to PTC Therapeutics for the work they are doing.  We look forward to their Phase 3 efficacy study.  It can’t happen soon enough.”

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Categories: NewsResearch


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