PTC Therapeutics

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Coordinates: 40°33′03″N 74°25′23″W / 40.550722°N 74.423071°W / 40.550722; -74.423071

PTC Therapeutics, Inc.
Industry Pharmaceuticals
Headquarters South Plainfield, New Jersey, United States

PTC Therapeutics is a US pharmaceutical company focused on the development of small molecule, orally administered treatments for orphan diseases.[1]

Since 2003, PTC has been working on a drug for Duchenne muscular dystrophy.[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]

As of May 2016, PTC portfolio includes small-molecule compounds for treatment of spinal muscular atrophy (RG7800 and RG7916) and Duchenne muscular dystrophy (Ataluren and another exon skipping compound).[4]


  1. ^ Bhutta, Sunya (December 2013). "Biopharma Blossoming in the Garden State". Biobusiness. Gen. Eng. Biotechnol. News. 33 (21). p. 15. 
  2. ^ Hamed SA (2006). "Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy". IDrugs : the investigational drugs journal. 9 (11): 783–9. PMID 17096300. 
  3. ^ Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". Retrieved 2016-05-14. 
  4. ^ "PTC Therapeutics | Pipeline". Retrieved 2016-05-14. 

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