(risdiplam), andPTChas leveraged the extensive learnings from theSMAprogram to broaden the platform to support discovery programs across numerous therapeutic areas includingneurodegenerativedisease, oncology and metabolism.PTChas also developed a ...
US enrollment for the phase 2 PIVOT-HD trial (NCT05358717) assessing PTC Therapeutics investigational agent PTC518 in patients with Huntington disease (HD) has been paused, according to the company, as the FDA continues to seek more data about the drug. Although stopped in the US, the stu...
PTC Therapeutics announced the initiation of a phase 1 clinical trial to evaluate PTC518 in healthy volunteers. PTC518 was identified from PTC's splicing platform and is being developed for the treatment of Huntington's disease (HD). PTC518 is an orally bioavailable molecule with broad central n...