Business
PTC Therapeutics to Host PTC518 Huntington Disease Deep Dive Webinar
SOUTH PLAINFIELD, N.J., April 7, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webinar in its deep dive series discussing the PTC518
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"SOUTH PLAINFIELD, N.J., April 7, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webinar in its deep dive series discussing the PTC518 Huntington disease (HD) program on Thursday, April 15 at 9 a.m. E.T. \nThe Huntington Disease Deep Dive will provide an overview of PTC's splicing platform and the identification of molecules that have selectivity and specificity. PTC will highlight its Huntington program and the potential advantages of an orally bioavailable compound. PTC will discuss in detail the splicing modifier, PTC518, that is in an ongoing Phase 1 clinical trial. In preclinical studies, PTC518 has demonstrated broad biodistribution that uniformly reaches all regions of the brain and periphery and has shown reduction in Huntingtin protein (HTT) levels with a 1:1 correlation between blood and brain. PTC will also present preliminary results from the Phase 1 study of PTC518. \nThe webinar can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the webinar and providing the passcode 5190575. A live, listen-only webcast can be accessed on the Events and Presentations page under the investor relations section of PTC Therapeutics' website at www.ptcbio.com. A webcast replay will be available approximately two hours after completion of the webinar and will be archived for 30 days following the webinar.\nAbout Huntington DiseaseHuntington disease (HD) is a rare, inherited disease that causes the progressive degeneration of nerve cells in the brain, impacting a person's functional abilities.1 While HD can present at any age, it is most prevalent in people aged 30 – 50, and it affects approximately 45,000 people in the United States.2,3 HD is caused by a mutation in the huntingtin gene, which is responsible for creating huntingtin protein (HTT).3 HTT protein is large and seems to have many functions, especially as the brain develops before birth, but it is not completely understood.3 As time progresses, the mutated huntingtin protein forms clumps in the brain cells, resulting in damaged cells and eventually cell death.3 \nAbout PTC518PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequ...