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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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Clinical Trials
Financial
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Company Revenue
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*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
,
limb-girdle muscular dystrophies
ELADOCAGENE EXUPARVOVEC
argininosuccinic aciduria
Sepiapterin
gastroparesis
,
phenylketonurias
Vatiquinone
ataxia
,
mitochondrial diseases
,
friedreich ataxia
,
leigh disease
,
melas syndrome
,
diffuse cerebral sclerosis of schilder
,
drug resistant epilepsy
,
merrf syndrome
,
seizures
,
status epilepticus
,
nerve degeneration
,
mitochondrial encephalomyopathies
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
Gilvetmab
colorectal neoplasms
,
squamous cell neoplasms
,
stomach neoplasms
,
esophageal neoplasms
,
neoplasm metastasis
,
sarcoma
,
squamous cell carcinoma
,
merkel cell carcinoma
,
endometrial neoplasms
,
hepatocellular carcinoma
,
head and neck neoplasms
,
pancreatic ductal carcinoma
,
colonic neoplasms
,
rectal neoplasms
,
triple negative breast neoplasms
,
mycoses
,
mycosis fungoides
,
digestive system neoplasms
,
gastrointestinal stromal tumors
,
uterine cervical neoplasms
,
small cell lung carcinoma
,
pancreatic neoplasms
,
bile duct neoplasms
,
liver neoplasms
,
rectal diseases
,
neoplasms
,
melanoma
,
urologic neoplasms
,
ovarian neoplasms
,
lung neoplasms
,
carcinoma
,
breast neoplasms
,
transitional cell carcinoma
,
ovarian epithelial carcinoma
,
adenocarcinoma
,
urologic diseases
,
kidney neoplasms
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
urinary bladder diseases
,
skin neoplasms
,
kidney diseases
,
urogenital neoplasms
,
neoplasms by site
,
glandular and epithelial neoplasms
,
non-small-cell lung carcinoma
,
renal cell carcinoma
,
squamous cell carcinoma of head and neck
,
urinary bladder neoplasms
,
non-muscle invasive bladder neoplasms
,
neoadjuvant therapy
Alfacalcidol
lung neoplasms
,
small cell lung carcinoma
,
hepatocellular carcinoma
,
liver neoplasms
,
lymphoma
,
large b-cell lymphoma diffuse
,
necrosis
,
malignant mesothelioma
Aspartame
osteoporosis
,
postmenopausal osteoporosis
Bevifimod
head and neck neoplasms
,
stroke
,
atherosclerosis
,
intracranial arteriosclerosis
,
fallopian tube neoplasms
,
neoplasms
,
squamous cell carcinoma of head and neck
,
adenocarcinoma
,
prostatic neoplasms
,
renal cell carcinoma
,
triple negative breast neoplasms
,
basal cell carcinoma
,
neoplasm metastasis
,
basal cell neoplasms
,
ovarian epithelial carcinoma
,
colorectal neoplasms
,
pancreatic neoplasms
,
hypersensitivity
,
peanut hypersensitivity
,
allergic rhinitis
,
rhinitis
,
chronic disease
,
healthy volunteers/patients
,
squamous cell carcinoma
,
merkel cell carcinoma
,
lung diseases
,
adenocarcinoma of lung
,
food hypersensitivity
,
milk hypersensitivity
,
smoldering multiple myeloma
,
monoclonal gammopathy of undetermined significance
,
obesity
,
bronchogenic carcinoma
,
bronchial neoplasms
,
paroxysmal hemoglobinuria
,
colonic neoplasms
,
malignant mesothelioma
,
mesothelioma
,
chronic renal insufficiency
,
diabetic nephropathies
,
carcinoma
,
rectal neoplasms
,
multiple myeloma
,
non-alcoholic fatty liver disease
,
non-small-cell lung carcinoma
,
melanoma
,
lung neoplasms
,
ovarian neoplasms
,
retinal diseases
,
follicular lymphoma
,
diabetic retinopathy
,
hemorrhage
,
b-cell lymphoma
,
rheumatoid arthritis
,
ascites
,
recurrence
,
type 2 diabetes mellitus
,
heterotopic ossification
,
myositis ossificans
,
vitreous hemorrhage
,
hodgkin disease
,
lymphoma
,
large b-cell lymphoma diffuse
,
vision disorders
,
eye diseases
,
blindness
,
hyperlipidemias
,
diabetes mellitus
,
b-cell lymphoma marginal zone
,
non-hodgkin lymphoma
,
macular degeneration
,
protein-losing enteropathies
,
nasal polyps
,
asthma
,
macular edema
,
hemophilia b
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
leukemia
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
covid-19
,
macular edema
,
uveitis
,
lymphoma
,
atopic dermatitis
,
follicular lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
non-small-cell lung carcinoma
,
inflammation
,
ischemic optic neuropathy
,
bullous pemphigoid
,
non-hodgkin lymphoma
,
lung neoplasms
,
exanthema
,
lupus nephritis
,
nephritis
,
neuromyelitis optica
,
nasal polyps
,
systemic lupus erythematosus
,
breast neoplasms
,
myasthenia gravis
,
pemphigus
,
multiple sclerosis
,
relapsing-remitting multiple sclerosis
,
chronic progressive multiple sclerosis
,
sclerosis
,
end stage liver disease
,
asthma
,
kidney transplantation
,
rheumatoid arthritis
,
arthritis
,
giant cell arteritis
,
arteritis
,
polymyalgia rheumatica
,
liver transplantation
,
juvenile arthritis
,
heart transplantation
,
kidney diseases
,
hypersensitivity
Lactams
cystic fibrosis
,
pain
Mk3475
carcinoma
,
colorectal neoplasms
,
pancreatic neoplasms
,
lung neoplasms
,
squamous cell carcinoma
,
squamous cell neoplasms
,
ovarian epithelial carcinoma
,
squamous cell carcinoma of head and neck
,
head and neck neoplasms
,
laryngeal diseases
,
camurati-engelmann syndrome
Solumedrol
atrial fibrillation
,
chronic renal insufficiency
,
chronic kidney failure
,
kidney transplantation
,
pulmonary fibrosis
,
secondary hyperparathyroidism
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