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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
,
fibrosis
,
cystic fibrosis
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
neuromuscular diseases
,
atrophic muscular disorders
Deflazacort
healthy volunteers/patients
,
renal insufficiency
,
liver diseases
,
hepatic insufficiency
,
muscular dystrophies
,
duchenne muscular dystrophy
,
limb-girdle muscular dystrophies
ELADOCAGENE EXUPARVOVEC
argininosuccinic aciduria
Vatiquinone
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
mitochondrial diseases
,
syndrome
,
leigh disease
,
brain diseases
,
epilepsy
,
diffuse cerebral sclerosis of schilder
,
drug resistant epilepsy
,
melas syndrome
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
Ademetionine
neoplasms
,
healthy volunteers/patients
,
renal insufficiency
,
liver diseases
,
hepatic insufficiency
,
aniridia
,
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
duchenne muscular dystrophy
,
muscular dystrophies
Aspartame
phenylketonurias
Bevifimod
healthy volunteers/patients
,
sarcoma
,
kaposi sarcoma
Ubidecarenone
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
syndrome
,
leigh disease
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Corticoids
neoplasms
,
duchenne muscular dystrophy
,
muscular dystrophies
PTC518
huntington disease
Mk3475
colorectal neoplasms
,
endometrial neoplasms
Solumedrol
healthy volunteers/patients
,
renal insufficiency
,
liver diseases
,
hepatic insufficiency
,
duchenne muscular dystrophy
,
muscular dystrophies
Docetaxol
neoplasms
1
2
>
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