| 词条 | PTC Therapeutics |
| 释义 |
| name = PTC Therapeutics, Inc. | logo = PTC Therapeutics.svg | type = Public | logo_size = 250px | traded_as = {{NASDAQ|PTCT}} Russell 2000 Component | industry = Pharmaceuticals | homepage = {{URL|ptcbio.com}} | location_city = South Plainfield, New Jersey | location_country = United States }}PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs that regulate gene expression by targeting post-translational control (PTC) mechanisms in orphan diseases.[1][2] In September 2009, PTC has entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases.[3] ProductsIn 2017, PTC acquired Emflaza[4] (deflazacort) from Marathon Pharmaceuticals. PTC also owns Translarna, (Ataluren) marketed for Duchenne muscular dystrophy.[5] Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.[6] Since 2003, PTC has been working on a drug for Duchenne muscular dystrophy.[7] {{As of|2016|05}}, PTC portfolio includes small-molecule compounds for treatment of spinal muscular atrophy (RG7916) and Duchenne muscular dystrophy (Ataluren and another exon skipping compound).[8] PipelineIn efforts to expand therapeutic offerings into other indications, PTC acquired Agilis Biotherapeutics in July 2018, a biotech company using DNA therapeutics to treat genetic disorders related to the central nervous system.[9] References1. ^{{Cite news|title = Biopharma Blossoming in the Garden State|date = December 2013|url = http://www.genengnews.com/gen-articles/biopharma-blossoming-in-the-garden-state/5075/|department = Biobusiness|last = Bhutta|first = Sunya|volume = 33|issue = 21|page = 15|work = Gen. Eng. Biotechnol. News}} 2. ^{{cite journal| doi=10.1016/j.nurx.2006.08.004 | pmid=17044149 | pmc=3593415 | title=Drug development in critical times | journal=NeuroRx | volume=3 | issue=4 | pages=540–3 | year=2006 | last1=Bain | first1=Lisa J }} 3. ^{{cite web|last=Bratulic |first=Anna |url=http://www.firstwordpharma.com/node/375238 |title=Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma |publisher=FirstWord Pharma |date=2009-09-02 |accessdate=2016-05-14}} 4. ^{{Cite news|url=https://seekingalpha.com/news/3251624-ptc-acquires-dmd-med-emflaza-marathon-pharma-190m|title=PTC acquires DMD med Emflaza from Marathon Pharma for up to $190M|last=House|first=SA Editor Douglas W.|date=2017-03-16|work=Seeking Alpha|access-date=2018-07-03|language=en-US}} 5. ^{{Cite news|url=https://www.nasdaq.com/article/in-the-spotlight-ptc-therapeutics-20180723-00195|title=In The Spotlight: PTC Therapeutics|date=2018-07-23|work=NASDAQ.com|access-date=2018-07-24|language=en-us}} 6. ^{{Cite news|url=https://www.nasdaq.com/article/in-the-spotlight-ptc-therapeutics-20180723-00195|title=In The Spotlight: PTC Therapeutics|date=2018-07-23|work=NASDAQ.com|access-date=2018-07-24|language=en-us}} 7. ^{{cite journal |author=Hamed SA |title=Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy |journal=IDrugs : the investigational drugs journal |volume=9 |issue=11 |pages=783–9 |year=2006 |pmid=17096300 |doi=}} 8. ^{{cite web|url=http://www.ptcbio.com/en/pipeline |title=PTC Therapeutics | Pipeline |publisher=Ptcbio.com |date= |accessdate=2016-05-14}} 9. ^{{Cite news|url=https://www.nasdaq.com/article/in-the-spotlight-ptc-therapeutics-20180723-00195|title=In The Spotlight: PTC Therapeutics|date=2018-07-23|work=NASDAQ.com|access-date=2018-07-25|language=en-us}} 10. ^ -->{{Cite journal | title = Firm Aims to Regulate RNA in Rare Diseases | url = http://www.genengnews.com/articles/chitem.aspx?aid=2869 | year = 2009 | author = Schneider, Ilene | journal = Genetic Engineering & Biotechnology News | page = 14 | volume = 29 | issue = 8 | accessdate = 2009-04-25 | postscript = }} External links
4 : Biotechnology|Pharmaceutical companies based in New Jersey|Companies based in Middlesex County, New Jersey|South Plainfield, New Jersey |
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