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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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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
Sepiapterin
gastroparesis
,
phenylketonurias
Vatiquinone
tic disorders
,
tourette syndrome
,
tics
,
alzheimer disease
,
dementia
,
muscular diseases
,
rett syndrome
,
ataxia
,
inborn genetic diseases
,
retinal diseases
,
homocystinuria
,
syndrome
,
inborn errors lipid metabolism
,
noise-induced hearing loss
,
angelman syndrome
,
autistic disorder
,
autism spectrum disorder
,
cerebellar ataxia
,
parkinson disease
,
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
,
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
Gilvetmab
neoplasms
,
squamous cell carcinoma of head and neck
,
colorectal neoplasms
,
glioblastoma
,
esophageal squamous cell carcinoma
,
astrocytoma
,
hepatocellular carcinoma
,
liver neoplasms
,
prostatic neoplasms
,
castration-resistant prostatic neoplasms
,
sarcoma
,
small cell lung carcinoma
,
malignant mesothelioma
,
neuroendocrine tumors
,
neuroendocrine carcinoma
,
squamous cell neoplasms
,
mesothelioma
,
colonic neoplasms
,
spinocerebellar degenerations
,
carcinoma
,
neoplasm metastasis
,
adenocarcinoma
Alfacalcidol
primary ovarian insufficiency
,
female infertility
,
infertility
,
ovulation induction
,
osteoporosis
,
postmenopausal osteoporosis
,
pregnancy
Aspartame
substance-related disorders
,
breakthrough pain
,
neoplasms
,
dyspnea
Bevifimod
macular edema
,
macular degeneration
,
choroidal neovascularization
,
refractive errors
,
myopia
,
astigmatism
,
hyperopia
,
contact lenses
,
hypertension
,
ocular hypertension
,
glaucoma
,
open-angle glaucoma
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
crohn disease
,
energy metabolism
,
infections
,
hiv infections
,
acquired immunodeficiency syndrome
,
atherosclerosis
,
kidney transplantation
,
delayed graft function
,
prostatic neoplasms
,
respiratory aspiration
,
autoimmune diseases
,
allergic rhinitis perennial
,
hemorrhage
,
hypereosinophilic syndrome
,
eosinophilia
,
syndrome
,
smoking
,
cardiovascular diseases
,
churg-strauss syndrome
,
granulomatosis with polyangiitis
,
systemic vasculitis
,
asthma
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
,
systemic lupus erythematosus
,
allergic rhinitis
,
rhinitis
,
thrombocytopenic purpura idiopathic
,
thrombocytopenia
,
inflammation
,
allergic rhinitis seasonal
,
emphysema
,
bronchitis
,
pulmonary eosinophilia
,
atopic dermatitis
,
pruritus
,
nasal polyps
,
fibrosis
,
cystic fibrosis
,
thrombocytopenic purpura
,
purpura
,
pneumonia
,
tobacco use disorder
,
tobacco smoking
Lactams
diabetes mellitus
,
autoimmune diseases
,
drug interactions
,
multiple sclerosis
,
tuberculosis
,
pulmonary tuberculosis
,
pharyngitis
,
tonsillitis
,
pneumonia
,
chronic bronchitis
,
sinusitis
,
bronchitis
,
bacterial pneumonia
Mk3475
head and neck neoplasms
,
squamous cell carcinoma of head and neck
,
lung neoplasms
,
neoplasms
,
carcinoma
,
stomach neoplasms
,
esophageal neoplasms
,
adenocarcinoma
,
non-hodgkin lymphoma
,
lymphoma
,
hodgkin disease
,
large-cell lymphoma anaplastic
,
t-cell lymphoma peripheral
,
ovarian neoplasms
,
squamous cell neoplasms
,
adenocarcinoma of lung
,
brain neoplasms
,
non-small-cell lung carcinoma
,
breast neoplasms
,
endometrial neoplasms
,
neoplasm metastasis
,
uterine neoplasms
,
urinary bladder neoplasms
,
transitional cell carcinoma
Solumedrol
uveitis
1
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