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PTC Therapeutics
PTC is a New Jersey rare-disease company, known for Duchenne and spinal muscular atrophy medicines, whose tubulin-binding drug unesbulin went into a phase 3 trial in leiomyosarcoma.
Also called: PTC Therapeutics, Inc.PTC
Overview
PTC Therapeutics, founded in 1998 and headquartered in South Plainfield, New Jersey, develops small molecules that modulate RNA and protein production, with approved medicines in Duchenne muscular dystrophy, spinal muscular atrophy and phenylketonuria. Its oncology programme unesbulin (PTC596), which binds tubulin and lowers BMI1, was taken into the phase 3 SUNRISE-LMS trial with dacarbazine in leiomyosarcoma. Its trials are registered on ClinicalTrials.gov under PTC as lead sponsor.
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Sources
Every source this page cites, grouped by who published it. Grouping is read from the web address, not assigned by hand.
ClinicalTrials.gov · 1
ptcbio.com · 1
wikidata.org · 1
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