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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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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
,
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
,
neuromuscular diseases
,
atrophic muscular disorders
,
chronic renal insufficiency
,
postoperative pain
,
trismus
,
impacted tooth
,
kidney diseases
,
iga glomerulonephritis
,
immunosuppression therapy
,
asthma
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
ovarian neoplasms
,
kidney neoplasms
,
leukemia
,
myelodysplastic syndromes
,
myeloid leukemia acute
,
hematologic diseases
,
myelomonocytic leukemia chronic
,
myeloid leukemia
,
myelomonocytic leukemia juvenile
,
preleukemia
,
bone marrow diseases
,
syndrome
,
gastrointestinal stromal tumors
,
glioblastoma
,
multiple myeloma
,
plasma cell neoplasms
,
alveolar soft part sarcoma
,
chordoma
,
thymoma
,
microsatellite instability
,
thymus neoplasms
,
cutaneous malignant melanoma
,
neoplasms
,
carcinoma
,
non-small-cell lung carcinoma
,
colorectal neoplasms
,
triple negative breast neoplasms
,
hepatocellular carcinoma
,
renal cell carcinoma
,
liver neoplasms
,
breast neoplasms
,
lung neoplasms
,
lymphoma
,
ovarian epithelial carcinoma
,
sarcoma
,
squamous cell carcinoma of head and neck
,
anus neoplasms
,
small cell lung carcinoma
,
adenocarcinoma
,
squamous cell carcinoma
,
squamous cell neoplasms
,
mesothelioma
,
pancreatic ductal carcinoma
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
pancreatic neoplasms
,
esophageal squamous cell carcinoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
prostatic neoplasms
,
castration-resistant prostatic neoplasms
,
neuroendocrine tumors
,
malignant mesothelioma
,
urologic neoplasms
,
thyroid neoplasms
,
esophageal neoplasms
,
uterine cervical neoplasms
,
cholangiocarcinoma
,
unknown primary neoplasms
,
adenocarcinoma of lung
,
clear cell adenocarcinoma
,
carcinosarcoma
,
uveal neoplasms
,
adenomyoepithelioma
,
uveal melanoma
,
hematologic neoplasms
,
anaplastic thyroid carcinoma
,
neuroendocrine carcinoma
,
melanoma
,
skin neoplasms
Alfacalcidol
melanoma
,
healthy volunteers/patients
,
herpes zoster
,
chickenpox
,
immunosenescence
,
pancreatic neoplasms
,
osteosarcoma
,
prostatic neoplasms
,
hepatitis b
,
chronic hepatitis b
,
hepatitis
,
chronic hepatitis
,
hepatitis a
Aspartame
recreational drug use
Bevifimod
clostridium infections
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
myelodysplastic syndromes
,
myeloid leukemia acute
,
recurrence
,
precursor cell lymphoblastic leukemia-lymphoma
,
neoplasm metastasis
,
biphenotypic leukemia acute
,
polymyalgia rheumatica
,
t-cell lymphoma
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
ovarian neoplasms
,
atopic dermatitis
,
ulcerative colitis
,
covid-19
,
arteritis
,
takayasu arteritis
,
aortic arch syndromes
,
giant cell arteritis
,
crohn disease
,
eczema
,
dermatitis
,
panuveitis
,
colitis
,
ulcer
Lactams
neoplasms
,
recurrence
,
non-hodgkin lymphoma
,
lymphoma
,
b-cell chronic lymphocytic leukemia
,
macular degeneration
,
wet macular degeneration
,
choroidal effusions
,
infections
,
communicable diseases
,
bacterial infections
,
methicillin-resistant staphylococcus aureus
,
staphylococcal skin infections
,
infectious skin diseases
,
bacterial skin diseases
,
cellulitis
Mk3475
leukemia
,
hematologic diseases
,
b-cell chronic lymphocytic leukemia
,
bone marrow diseases
,
colorectal neoplasms
,
colonic neoplasms
,
colonic diseases
,
neoplasms
,
squamous cell carcinoma of head and neck
Solumedrol
macular edema
,
retinal diseases
,
diabetic retinopathy
,
uveitis
,
anterior uveitis
,
iridocyclitis
,
inflammation
,
cataract
,
edema
,
corneal edema
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