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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:
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
,
mitochondrial diseases
,
rett syndrome
,
muscular diseases
,
syndrome
,
inborn errors lipid metabolism
,
noise-induced hearing loss
,
angelman syndrome
,
autistic disorder
,
autism spectrum disorder
,
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
parkinson disease
,
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
renal cell carcinoma
,
neoplasms
,
non-small-cell lung carcinoma
,
urinary bladder neoplasms
,
melanoma
,
oncogenes
,
urologic neoplasms
,
carcinoma
,
transitional cell carcinoma
Alfacalcidol
major depressive disorder
,
depressive disorder
,
prostatic hyperplasia
,
osteoporosis
Aspartame
healthy volunteers/patients
,
covid-19
,
obesity
,
adiposity
,
stroke
,
muscle spasticity
,
shoulder pain
Bevifimod
pharmacokinetics
,
inflammatory breast neoplasms
,
carcinoma
,
renal cell carcinoma
,
communicable diseases
,
pneumococcal infections
,
immune system diseases
,
triple negative breast neoplasms
,
diabetic foot
,
ulcer
,
foot ulcer
,
glioblastoma
,
gliosarcoma
,
astrocytoma
,
oligodendroglioma
,
small cell lung carcinoma
,
neoplasm metastasis
,
stomach neoplasms
,
esophageal neoplasms
,
hodgkin disease
,
adenocarcinoma
,
precursor cell lymphoblastic leukemia-lymphoma
,
melanoma
,
bronchiolitis obliterans syndrome
,
meningitis
,
plasma cell neoplasms
,
squamous cell carcinoma of head and neck
,
ovarian epithelial carcinoma
,
human influenza
,
fallopian tube neoplasms
,
biliary tract neoplasms
,
prolymphocytic leukemia
,
pancreatic neoplasms
,
tuberculosis
,
hepatocellular carcinoma
,
liver neoplasms
,
head and neck neoplasms
,
pancreatic ductal carcinoma
,
induction chemotherapy
,
lung diseases
,
adenocarcinoma of lung
,
gestational diabetes
,
pregnancy in diabetics
,
coronary disease
,
large b-cell lymphoma diffuse
,
brain neoplasms
,
mucopolysaccharidoses
,
mucopolysaccharidosis i
,
follicular lymphoma
,
hematopoietic stem cell transplantation
,
peripheral arterial disease
,
atherosclerosis
,
intermittent claudication
,
peripheral vascular diseases
,
mumps
,
measles
,
chickenpox
,
rubella
,
meningococcal meningitis
,
haemophilus infections
,
cutaneous malignant melanoma
,
esophageal squamous cell carcinoma
,
urinary bladder neoplasms
,
diarrhea
,
rectal neoplasms
,
congenital abnormalities
,
megakaryoblastic leukemia acute
,
monocytic leukemia acute
,
myelomonocytic leukemia acute
,
erythroblastic leukemia acute
,
hypersensitivity
,
peanut hypersensitivity
,
squamous cell carcinoma
,
squamous cell neoplasms
,
prolymphocytic leukemia t-cell
,
allergic rhinitis
,
rhinitis
,
acquired immunodeficiency syndrome
,
sezary syndrome
,
mycosis fungoides
,
t-cell lymphoma cutaneous
,
pulmonary alveolar proteinosis
,
preleukemia
,
t-cell lymphoma peripheral
,
laryngeal neoplasms
,
emphysema
,
alpha 1-antitrypsin deficiency
,
breast neoplasms
,
colorectal neoplasms
,
multiple myeloma
,
non-small-cell lung carcinoma
,
leukemia
,
myeloid leukemia acute
,
b-cell chronic lymphocytic leukemia
,
lymphoma
,
recurrence
,
lung neoplasms
,
myelodysplastic syndromes
,
diphtheria
,
ovarian neoplasms
,
whooping cough
,
tetanus
,
graft vs host disease
,
meningococcal infections
,
poliomyelitis
,
healthy volunteers/patients
,
hiv infections
,
hematologic neoplasms
,
hemophilia b
,
pneumococcal pneumonia
,
hepatitis
,
haemophilus influenzae type b
,
infections
,
hepatitis b
,
myeloid leukemia
,
thrombosis
,
venous thrombosis
,
rheumatoid arthritis
,
ascites
,
bcr-abl positive chronic myelogenous leukemia
,
aplastic anemia
,
thrombotic thrombocytopenic purpura
,
insulinoma
,
neuroendocrine tumors
,
carcinoid tumor
,
vipoma
,
paraneoplastic syndromes
,
islet cell carcinoma
,
islet cell adenoma
,
gastrinoma
,
glucagonoma
,
somatostatinoma
,
pulmonary embolism
,
embolism
,
motor activity
,
st elevation myocardial infarction
,
glucose intolerance
,
hematologic diseases
,
inborn genetic diseases
,
blood coagulation disorders
,
x-linked genetic diseases
,
hemorrhagic disorders
,
inherited blood coagulation disorders
,
coagulation protein disorders
,
fetal growth retardation
,
thromboembolism
,
noonan syndrome
,
bacterial meningitis
,
tonsillitis
,
myeloproliferative disorders
,
chronic inflammatory demyelinating polyradiculoneuropathy
,
anemia
,
type 2 diabetes mellitus
,
type 1 diabetes mellitus
,
glycogen storage disease type ii
,
neoplasms
,
diabetes mellitus
,
prostatic neoplasms
,
non-hodgkin lymphoma
,
multiple sclerosis
,
hemophilia a
,
glycogen storage disease type iii
,
relapsing-remitting multiple sclerosis
,
covid-19
,
hyperglycemia
,
graft rejection
,
chronic kidney failure
,
insulin resistance
,
renal insufficiency
,
kidney transplantation
,
autologous transplantation
,
acute coronary syndrome
,
myocardial infarction
,
infarction
,
liver diseases
,
liver transplantation
,
psychology rejection
,
atrial fibrillation
,
pneumonia
,
hypertension
,
neutropenia
,
inflammation
,
heartburn
,
colitis
,
nasal polyps
,
asthma
,
inferior wall myocardial infarction
,
kidney diseases
,
coronary artery disease
,
immunosuppression therapy
,
fabry disease
,
anaphylaxis
,
glycogen storage disease
,
pregnancy
,
lymphoproliferative disorders
,
paraproteinemias
,
heart transplantation
,
reperfusion injury
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
cutaneous lupus erythematosus
,
b-cell lymphoma
,
non-hodgkin lymphoma
,
lymphoma
,
large b-cell lymphoma diffuse
,
graft vs host disease
,
bronchiolitis obliterans syndrome
,
recurrence
,
covid-19
,
coronavirus infections
,
severe acute respiratory syndrome
,
virus diseases
,
coronaviridae infections
,
nidovirales infections
,
rna virus infections
Lactams
pneumonia
Mk3475
renal cell carcinoma
,
neoplasms
,
non-small-cell lung carcinoma
,
urinary bladder neoplasms
,
melanoma
,
oncogenes
Solumedrol
atrial fibrillation
,
chronic renal insufficiency
,
chronic kidney failure
,
kidney transplantation
,
pulmonary fibrosis
,
secondary hyperparathyroidism
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