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PTC Therapeutics
PTC Therapeutics
(PTCT)
PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.
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Financial
Marketed Drugs
Clinical Trials
Financial
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*Currency in USD
Company Revenue
Quarterly
Quarterly
Annual
Annual
Projections
*Quarterly sales USD (in millions)
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Revenue by Drug
Marketed Drugs
Therapeutic Areas:
All
Classification:
All
Include Generics:
Trade Name
Drug Name
Approved
Patent Expires
Indication
Emflaza
Deflazacort
2017-02-09
Inflammation
,
Duchenne muscular dystrophy
Translarna
Ataluren
2014-07-31
Duchenne muscular dystrophy
Upstaza
Eladocagene exuparvovec
2022-07-18
Inborn errors amino acid metabolism
Clinical Trials
Therapeutic Areas:
All
Classification:
All
Approval:
All
Status:
All
Phases:
All
Drug Name
Phase 1
Phase 2
Phase 3
Phase 4
Indication
Ataluren
healthy volunteers/patients
,
aniridia
,
hemophilia a
,
hemophilia b
,
colorectal neoplasms
,
endometrial neoplasms
,
epilepsy
,
myoclonic epilepsies
,
inborn errors metabolism
,
inborn errors amino acid metabolism
,
duchenne muscular dystrophy
,
muscular dystrophies
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
neuromuscular diseases
,
atrophic muscular disorders
,
fibrosis
,
cystic fibrosis
Deflazacort
healthy volunteers/patients
,
renal insufficiency
,
liver diseases
,
hepatic insufficiency
,
muscular dystrophies
,
duchenne muscular dystrophy
,
proteinuria
,
neuromuscular diseases
,
membranous glomerulonephritis
,
aspergillosis
,
allergic bronchopulmonary aspergillosis
,
pulmonary aspergillosis
,
limb-girdle muscular dystrophies
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
atrophic muscular disorders
,
chronic renal insufficiency
,
postoperative pain
,
trismus
,
impacted tooth
,
hearing loss
,
otitis media with effusion
,
conductive hearing loss
,
kidney diseases
,
iga glomerulonephritis
,
immunosuppression therapy
,
asthma
Vatiquinone
tic disorders
,
tourette syndrome
,
tics
,
mitochondrial diseases
,
rett syndrome
,
muscular diseases
,
syndrome
,
inborn errors lipid metabolism
,
noise-induced hearing loss
,
angelman syndrome
,
autistic disorder
,
autism spectrum disorder
,
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
parkinson disease
,
optic nerve diseases
,
emergencies
,
hereditary optic atrophy leber
Emvododstat
neoplasms
,
breast neoplasms
,
central nervous system neoplasms
,
nervous system neoplasms
,
leukemia
,
myeloid leukemia acute
,
myeloid leukemia
,
sarcoma
,
kaposi sarcoma
,
neurofibromatosis 2
,
neurofibromatosis 1
,
neurofibromatoses
,
neurofibroma
,
covid-19
,
coronavirus infections
,
coronavirus
,
pneumonia
ELADOCAGENE EXUPARVOVEC
argininosuccinic aciduria
Unesbulin
glioma
,
glioblastoma
,
astrocytoma
,
oligodendroglioma
,
ovarian neoplasms
,
ovarian epithelial carcinoma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Sepiapterin
gastroparesis
,
phenylketonurias
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