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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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Revenue by Drug
Marketed Drugs
Therapeutic Areas:
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Classification:
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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
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
,
non-small-cell lung carcinoma
,
lung neoplasms
,
carcinoma
,
head and neck neoplasms
,
squamous cell carcinoma of head and neck
,
melanoma
,
squamous cell carcinoma
Alfacalcidol
ulcerative colitis
Aspartame
tuberculosis
,
neoplasms
,
myeloid leukemia acute
,
deficiency diseases
,
paraproteinemias
,
hiv infections
,
precursor cell lymphoblastic leukemia-lymphoma
,
breast neoplasms
,
immune system diseases
,
hematologic neoplasms
,
type 2 diabetes mellitus
,
uveitis
,
metabolic diseases
,
disease
,
glycogen storage disease
,
lipidoses
,
immunoglobulin g4-related disease
,
leukemia
,
myeloid leukemia
,
malaria
,
falciparum malaria
,
whooping cough
,
tetanus
,
diphtheria
,
tetany
,
polycystic kidney diseases
,
autosomal dominant polycystic kidney
,
arthrogryposis
,
recurrence
,
renal insufficiency
,
hematologic diseases
,
blood protein disorders
,
neoplasms by histologic type
,
prostatic neoplasms
,
circulating neoplastic cells
,
x-linked bulbo-spinal atrophy
,
heart transplantation
,
non-small-cell lung carcinoma
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
esophageal neoplasms
,
adenocarcinoma
,
neoplasm metastasis
,
aplastic anemia
,
multiple myeloma
,
hypercholesterolemia
,
meningococcal infections
,
plasma cell neoplasms
,
hemolytic anemia autoimmune
,
meningitis
,
meningococcal meningitis
,
stomach neoplasms
,
hemophilia a
,
kidney diseases
,
gaucher disease
,
hemophilia b
,
chronic renal insufficiency
,
hyperphosphatemia
,
multiple sclerosis
,
sclerosis
,
homozygous familial hypercholesterolemia
,
hyperlipoproteinemia type ii
,
pharyngitis
,
tonsillitis
,
healthy volunteers/patients
,
asthma
,
glycogen storage disease type ii
,
atopic dermatitis
,
diabetes mellitus
,
insulin resistance
,
hyperglycemia
,
glycogen storage disease type iii
,
heartburn
,
non-hodgkin lymphoma
,
osteoporosis
,
postmenopausal osteoporosis
,
lymphoma
,
lymphoproliferative disorders
Bevifimod
healthy volunteers/patients
,
hiv-1
,
bcr-abl positive chronic myelogenous leukemia
,
type 2 diabetes mellitus
,
glandular and epithelial neoplasms
,
myoepithelioma
,
gata2 deficiency
,
central nervous system neoplasms
,
hiv-2
,
fallopian tube diseases
,
neurologic manifestations
,
sepsis
,
interdigitating dendritic cell sarcoma
,
myeloproliferative disorders
,
neoplasms by histologic type
,
adenosquamous carcinoma
,
chronic hepatitis c
,
astrocytoma
,
venous thromboembolism
,
thromboembolism
,
pharmacokinetics
,
plasmacytoma
,
behcet syndrome
,
aids-related complex
,
pleural effusion
,
respiratory tract neoplasms
,
bronchogenic carcinoma
,
thoracic neoplasms
,
malignant pleural effusion
,
pleural diseases
,
pleural neoplasms
,
immunosuppression therapy
,
small cell lung carcinoma
,
transitional cell carcinoma
,
ovarian neoplasms
,
non-hodgkin lymphoma
,
triple negative breast neoplasms
,
urologic neoplasms
,
squamous cell carcinoma
,
sarcoma
,
biliary tract neoplasms
,
malignant mesothelioma
,
large b-cell lymphoma diffuse
,
glioma
,
uterine cervical neoplasms
,
neoplasms by site
,
castration-resistant prostatic neoplasms
,
b-cell lymphoma
,
endometrial neoplasms
,
leukemia
,
squamous cell neoplasms
,
kaposi sarcoma
,
uveal neoplasms
,
uveal melanoma
,
hematologic neoplasms
,
t-cell lymphoma peripheral
,
t-cell lymphoma
,
b-cell chronic lymphocytic leukemia
,
chordoma
,
neuroendocrine tumors
,
gastrointestinal neoplasms
,
adenocarcinoma of lung
,
myeloid leukemia acute
,
cholangiocarcinoma
,
graft vs host disease
,
interstitial lung diseases
,
follicular lymphoma
,
merkel cell carcinoma
,
peritoneal neoplasms
,
spinocerebellar degenerations
,
osteosarcoma
,
graft rejection
,
psychology rejection
,
clear cell adenocarcinoma
,
nose neoplasms
,
neuroblastoma
,
medulloblastoma
,
ependymoma
,
ewing sarcoma
,
rhabdomyosarcoma
,
diffuse intrinsic pontine glioma
,
gliosarcoma
,
autoimmune diseases
,
multiple sclerosis
,
waldenstrom macroglobulinemia
,
extranodal nk-t-cell lymphoma
,
precursor cell lymphoblastic leukemia-lymphoma
,
burkitt lymphoma
,
female genital neoplasms
,
endometrioid carcinoma
,
leiomyosarcoma
,
carcinosarcoma
,
urogenital neoplasms
,
organ transplantation
,
neuroendocrine carcinoma
,
urinary bladder diseases
,
microsatellite instability
,
basal cell carcinoma
,
basal cell neoplasms
,
diffuse scleroderma
,
laryngeal diseases
,
laryngitis
,
heart transplantation
,
plant tumors
,
male breast neoplasms
,
b-cell leukemia
,
tertiary lymphoid structures
,
oropharyngeal neoplasms
,
fowlpox
,
prostatic diseases
,
non-muscle invasive bladder neoplasms
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
diabetic neuropathies
,
nephrotic syndrome
,
focal segmental glomerulosclerosis
,
lipoid nephrosis
,
bronchiolitis obliterans syndrome
,
diabetic nephropathies
,
kidney diseases
,
common variable immunodeficiency
,
large-cell lymphoma anaplastic
,
rhabdoid tumor
,
phenylketonurias
,
bile duct neoplasms
,
serous cystadenocarcinoma
,
papillary thyroid cancer
,
myositis
,
salivary gland neoplasms
,
genetic therapy
,
smoldering multiple myeloma
,
uterine neoplasms
,
large cell carcinoma
,
endometrial stromal tumors
,
small cell carcinoma
,
penile neoplasms
,
adrenocortical carcinoma
,
digestive system diseases
,
hypertension
,
inflammation
,
hepatoblastoma
,
wilms tumor
,
invasive hydatidiform mole
,
pruritus
,
exanthema
,
urticaria
,
bronchiolo-alveolar adenocarcinoma
,
drug-related side effects and adverse reactions
,
carcinoid tumor
,
hypersensitivity
,
thyroid neoplasms
,
precancerous conditions
,
bronchopulmonary dysplasia
,
tobacco smoking
,
b-cell lymphoma marginal zone
,
mantle-cell lymphoma
,
meningeal carcinomatosis
,
systemic scleroderma
,
patient preference
,
uveitis
,
oral leukoplakia
,
churg-strauss syndrome
,
microscopic polyangiitis
,
oligodendroglioma
,
myeloid leukemia
,
preleukemia
,
syndrome
,
ankylosing spondylitis
,
motor activity
,
inflammatory breast neoplasms
,
immunoglobulin g4-related disease
,
cerebral palsy
,
sarcoidosis
,
progressive multifocal leukoencephalopathy
,
gastrointestinal stromal tumors
,
male urogenital diseases
,
female urogenital diseases
,
female urogenital diseases and pregnancy complications
,
urogenital diseases
,
reactive arthritis
,
hereditary nonpolyposis colorectal neoplasms
,
disease
,
aplastic anemia
,
melanoma
,
neoplasms
,
non-small-cell lung carcinoma
,
colorectal neoplasms
,
neoplasm metastasis
,
squamous cell carcinoma of head and neck
,
prostatic neoplasms
,
head and neck neoplasms
,
breast neoplasms
,
pancreatic neoplasms
,
liver neoplasms
,
hepatocellular carcinoma
,
urinary bladder neoplasms
,
adenocarcinoma
,
brain neoplasms
,
stomach neoplasms
,
hodgkin disease
,
multiple myeloma
,
recurrence
,
colonic neoplasms
,
glioblastoma
,
rectal neoplasms
,
esophageal neoplasms
,
carcinoma
,
lymphoma
,
skin neoplasms
,
cutaneous malignant melanoma
,
pancreatic ductal carcinoma
,
fallopian tube neoplasms
,
systemic lupus erythematosus
,
myelodysplastic syndromes
,
plasma cell neoplasms
,
mesothelioma
,
ovarian epithelial carcinoma
,
psoriasis
,
arthritis
,
esophageal squamous cell carcinoma
,
ulcerative colitis
,
juvenile arthritis
,
psoriatic arthritis
,
sjogren's syndrome
,
lung diseases
,
crohn disease
,
lupus nephritis
,
giant cell arteritis
,
respiratory tract diseases
,
granulomatosis with polyangiitis
,
anti-neutrophil cytoplasmic antibody-associated vasculitis
,
hyperlipidemias
,
polymyalgia rheumatica
,
vascular diseases
,
vasculitis
,
systemic vasculitis
,
disease progression
,
pathologic complete response
,
anemia
,
rheumatoid arthritis
,
renal cell carcinoma
,
lung neoplasms
,
hiv infections
,
kidney transplantation
,
acquired immunodeficiency syndrome
,
hiv
,
kidney neoplasms
,
delayed graft function
,
chronic kidney failure
,
coronary artery disease
,
myocarditis
,
hiv-associated lipodystrophy syndrome
,
epstein-barr virus infections
,
ischemia
,
transplantation
,
mitochondrial diseases
,
lipodystrophy
,
lymphocytosis
,
parkinson disease
,
necrosis
,
cardiomyopathies
,
dilated cardiomyopathy
,
colitis
,
ulcer
,
alzheimer disease
,
progressive supranuclear palsy
,
paralysis
,
obesity
,
diabetes mellitus
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
healthy volunteers/patients
,
breast neoplasms
,
renal cell carcinoma
,
brain neoplasms
,
ovarian neoplasms
,
pancreatic neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
kidney neoplasms
,
atopic dermatitis
,
eczema
,
dermatitis
,
immune system diseases
,
inflammation
,
bronchial diseases
,
hypersensitivity
,
immediate hypersensitivity
,
respiratory hypersensitivity
,
dyspnea
,
colorectal neoplasms
,
nasal polyps
,
gastroesophageal reflux
,
bullous pemphigoid
,
churg-strauss syndrome
,
nasopharyngeal neoplasms
,
eosinophilic esophagitis
,
peptic esophagitis
,
esophagitis
,
asthma
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
,
systemic lupus erythematosus
,
pulmonary eosinophilia
,
eosinophilia
,
respiratory tract diseases
,
respiration disorders
,
emphysema
,
respiratory sounds
,
disease progression
,
allergic rhinitis
,
rhinitis
,
allergic rhinitis perennial
,
hypercholesterolemia
,
metabolic bone diseases
,
osteoporosis
,
bone fractures
,
osteoporotic fractures
Lactams
pharmacokinetics
,
crohn disease
,
inflammatory bowel diseases
,
syndrome
,
irritable bowel syndrome
,
abdominal pain
,
escherichia coli infections
,
bacillary dysentery
,
clostridium infections
,
diarrhea
,
hepatic encephalopathy
,
liver cirrhosis
,
fibrosis
,
hepatic insufficiency
,
hepatitis c
,
liver diseases
,
liver failure
,
brain diseases
,
hiv
,
pouchitis
,
bacterial infections
,
gastroenteritis
,
celiac disease
,
chronic hepatitis c
Mk3475
non-small-cell lung carcinoma
,
lung neoplasms
Solumedrol
hyperlipoproteinemia type i
1
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