PTC Therapeutics

E561093

PTC Therapeutics is a biopharmaceutical company focused on developing small-molecule and RNA-based therapies for rare genetic disorders and other serious diseases.

All labels observed (1)

Label Occurrences
PTC Therapeutics canonical 5

How this entity was disambiguated

Statements (47)

Predicate Object
instanceOf biopharmaceutical company
public company
collaboratesWith Roche
SMA Foundation
conducts clinical trials
country United States
develops RNA-based therapies
small-molecule therapies
engagesIn commercialization of medicines
drug discovery
preclinical development
focusesOn rare genetic disorders
serious diseases
foundedBy Stuart W. Peltz
hasBusinessModel research and development of proprietary therapeutics
hasDrug Emflaza
Evrysdi
Translarna
Upstaza
hasDrugIndication Duchenne muscular dystrophy
aromatic L-amino acid decarboxylase deficiency
spinal muscular atrophy
hasEmployeeType clinical development professionals
regulatory affairs professionals
research scientists
hasKeyPerson Matthew B. Klein
Stuart W. Peltz
hasMarket global rare disease market
hasTherapeuticArea central nervous system disorders
genetic diseases
metabolic disorders
neuromuscular disorders
oncology
headquartersLocation South Plainfield, New Jersey, United States
industry biotechnology
pharmaceuticals
regulatoryRegion European Union
Latin America
United States
researchFocus nonsense mutation readthrough
targetsPatientPopulation patients with rare diseases
pediatric patients
tradedAs NASDAQ:PTCT
usesModality RNA splicing modulation
gene therapy
small molecules
website https://www.ptcbio.com

How these facts were elicited

Referenced by (5)

Full triples — surface form annotated when it differs from this entity's canonical label.

risdiplam developedBy PTC Therapeutics
Evrysdi hasDeveloper PTC Therapeutics
FIREFISH developedWith PTC Therapeutics
SUNFISH developedBy PTC Therapeutics
JEWELFISH collaborator PTC Therapeutics