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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
,
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
,
kidney diseases
,
limb-girdle muscular dystrophies
,
glomerulonephritis
,
membranous glomerulonephritis
,
aspergillosis
,
allergic bronchopulmonary aspergillosis
,
pulmonary aspergillosis
,
chronic renal insufficiency
,
chronic kidney failure
,
postoperative pain
,
trismus
,
impacted tooth
,
proteinuria
,
iga glomerulonephritis
,
immunosuppression therapy
,
asthma
ELADOCAGENE EXUPARVOVEC
argininosuccinic aciduria
Vatiquinone
tourette syndrome
,
muscular diseases
,
inborn genetic diseases
,
homocystinuria
,
inborn errors lipid metabolism
,
hearing loss
,
noise-induced hearing loss
,
deafness
,
autistic disorder
,
autism spectrum disorder
,
pervasive child development disorders
,
parkinson disease
,
rett syndrome
,
syndrome
,
mitochondrial diseases
,
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
leigh disease
,
brain diseases
,
epilepsy
,
diffuse cerebral sclerosis of schilder
,
drug resistant epilepsy
,
melas syndrome
,
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
urinary bladder neoplasms
,
astrocytoma
,
glandular and epithelial neoplasms
,
myoepithelioma
,
hepatitis c
,
hepatitis
,
hepatitis a
,
neoplasms by histologic type
,
urinary bladder diseases
,
hodgkin disease
,
myelodysplastic syndromes
,
myeloid leukemia acute
,
neoplasms
,
pancreatic neoplasms
,
adenocarcinoma
,
colorectal neoplasms
,
glioblastoma
,
small cell lung carcinoma
,
recurrence
,
skin neoplasms
,
lymphoma
,
cutaneous malignant melanoma
,
prostatic neoplasms
,
glioma
,
transitional cell carcinoma
,
liver neoplasms
,
pancreatic ductal carcinoma
,
colonic neoplasms
,
urologic neoplasms
,
endometrial neoplasms
,
hepatocellular carcinoma
,
neoplasms by site
,
stomach neoplasms
,
chordoma
,
female genital neoplasms
,
sarcoma
,
cholangiocarcinoma
,
gliosarcoma
,
rectal neoplasms
,
kidney neoplasms
,
follicular lymphoma
,
squamous cell neoplasms
,
multiple myeloma
,
plasma cell neoplasms
,
endometrioid carcinoma
,
biliary tract neoplasms
,
neuroendocrine tumors
,
adenocarcinoma of lung
,
nose neoplasms
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
oropharyngeal neoplasms
,
prostatic diseases
,
diffuse intrinsic pontine glioma
,
hematologic neoplasms
,
uterine cervical neoplasms
,
bile duct neoplasms
,
genetic therapy
,
serous cystadenocarcinoma
,
smoldering multiple myeloma
,
carcinosarcoma
,
mullerian mixed tumor
,
urogenital neoplasms
,
small cell carcinoma
,
neuroendocrine carcinoma
,
penile neoplasms
,
adrenocortical carcinoma
,
gastrointestinal neoplasms
,
mesothelioma
,
drug-related side effects and adverse reactions
,
mouth neoplasms
,
basal cell carcinoma
,
basal cell neoplasms
,
bronchopulmonary dysplasia
,
tobacco smoking
,
b-cell lymphoma marginal zone
,
mantle-cell lymphoma
,
waldenstrom macroglobulinemia
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
burkitt lymphoma
,
oligodendroglioma
,
merkel cell carcinoma
,
hereditary nonpolyposis colorectal neoplasms
,
adenoma
,
syndrome
,
non-hodgkin lymphoma
,
microsatellite instability
,
melanoma
,
non-small-cell lung carcinoma
,
carcinoma
,
lung neoplasms
,
renal cell carcinoma
,
squamous cell carcinoma of head and neck
,
neoplasm metastasis
,
breast neoplasms
,
squamous cell carcinoma
,
head and neck neoplasms
,
brain neoplasms
,
triple negative breast neoplasms
,
ovarian neoplasms
,
fallopian tube neoplasms
,
ovarian epithelial carcinoma
Aspartame
healthy volunteers/patients
,
alzheimer disease
,
female genital neoplasms
,
melanoma
,
hepatitis b
,
chronic hepatitis b
,
depression
,
major depressive disorder
,
depressive disorder
,
lung neoplasms
,
triple negative breast neoplasms
,
pancreatic neoplasms
,
neoplasm metastasis
,
coronary disease
,
coronary artery disease
,
myocardial ischemia
,
heart diseases
,
chronic hepatitis
,
sickle cell anemia
,
adenocarcinoma
,
sarcoma
,
glioma
,
neuroblastoma
,
precursor cell lymphoblastic leukemia-lymphoma
,
parkinson disease
,
autistic disorder
,
autism spectrum disorder
,
pervasive child development disorders
,
rectal neoplasms
,
covid-19
,
pneumonia
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
exanthema
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
stomach neoplasms
,
esophageal neoplasms
,
neoplasms
,
colorectal neoplasms
,
non-small-cell lung carcinoma
,
breast neoplasms
,
lupus nephritis
,
nephritis
,
large b-cell lymphoma diffuse
,
human influenza
,
anemia
,
leukemia
,
b-cell lymphoma
,
carcinoma
,
non-hodgkin lymphoma
,
myeloid leukemia acute
,
multiple sclerosis
,
sclerosis
,
hepatocellular carcinoma
,
colonic neoplasms
,
thrombocytopenic purpura idiopathic
,
thrombocytopenic purpura
,
purpura
,
liver cirrhosis
,
neuromyelitis optica
,
kidney diseases
,
membranous glomerulonephritis
,
chronic progressive multiple sclerosis
,
myeloid leukemia
,
nephrotic syndrome
,
nephrosis
,
lipoid nephrosis
,
recurrence
,
ovarian neoplasms
,
hemolysis
,
hemolytic anemia autoimmune
,
hemolytic anemia
,
hepatitis
,
hepatitis a
,
arthritis
,
hepatitis c
,
chronic hepatitis c
,
lymphoma
,
rheumatoid arthritis
,
juvenile arthritis
,
follicular lymphoma
,
syndrome
,
hiv infections
,
asthma
,
heart failure
,
myelodysplastic syndromes
,
preleukemia
,
acquired immunodeficiency syndrome
,
kidney transplantation
,
drug-related side effects and adverse reactions
,
heart disease risk factors
,
cognitive dysfunction
Bevifimod
fibrosis
,
cystic fibrosis
,
pulmonary fibrosis
Ubidecarenone
neoplasms
,
gastrointestinal neoplasms
,
lung neoplasms
,
neuroendocrine tumors
,
carcinoid tumor
,
paraneoplastic syndromes
,
islet cell adenoma
,
malignant carcinoid syndrome
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
,
edema
,
lymphoma
,
atopic dermatitis
,
eczema
,
dermatitis
,
ischemic optic neuropathy
,
follicular lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
non-small-cell lung carcinoma
,
lung neoplasms
,
non-hodgkin lymphoma
,
bullous pemphigoid
,
kidney diseases
,
inflammation
,
lupus nephritis
,
nephritis
,
arteritis
,
giant cell arteritis
,
polymyalgia rheumatica
,
systemic lupus erythematosus
,
neuromyelitis optica
,
nasal polyps
,
polyps
,
rhinosinusitis
,
breast neoplasms
,
hand-foot syndrome
,
myasthenia gravis
,
muscle weakness
,
pemphigus
,
sinusitis
,
multiple sclerosis
,
relapsing-remitting multiple sclerosis
,
chronic progressive multiple sclerosis
,
sclerosis
,
asthma
,
kidney transplantation
,
arthritis
,
rheumatoid arthritis
,
liver transplantation
,
juvenile arthritis
,
heart transplantation
,
hypersensitivity
Mk3475
neoplasms
,
neoplasm metastasis
,
carcinoma
,
squamous cell carcinoma of head and neck
,
malignant mesothelioma
,
mesothelioma
,
squamous cell carcinoma
,
colorectal neoplasms
,
hepatocellular carcinoma
,
stomach neoplasms
,
gastrointestinal neoplasms
,
liver neoplasms
,
esophageal squamous cell carcinoma
,
lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
non-small-cell lung carcinoma
,
lung neoplasms
Solumedrol
b-cell lymphoma
,
prostatic neoplasms
,
non-hodgkin lymphoma
,
large b-cell lymphoma diffuse
,
focal segmental glomerulosclerosis
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
interstitial nephritis
,
rheumatoid arthritis
,
arthritis
,
lymphoma
,
nephritis
,
systemic lupus erythematosus
,
lupus nephritis
,
vasculitis
,
granulomatosis with polyangiitis
,
microscopic polyangiitis
,
systemic vasculitis
,
anti-neutrophil cytoplasmic antibody-associated vasculitis
Docetaxol
urethral neoplasms
,
transitional cell carcinoma
,
urologic neoplasms
,
small cell carcinoma
,
small cell lung carcinoma
,
neuroendocrine tumors
,
neuroendocrine carcinoma
,
kidney neoplasms
,
ureteral neoplasms
,
colorectal neoplasms
,
neoplasms
,
castration-resistant prostatic neoplasms
,
recurrence
,
triple negative breast neoplasms
,
urinary bladder neoplasms
,
sarcoma
,
laryngeal neoplasms
,
laryngeal diseases
,
hepatocellular carcinoma
,
liver neoplasms
,
uterine cervical neoplasms
,
cholangiocarcinoma
,
gallbladder neoplasms
,
maxillary sinus neoplasms
,
neoplasm metastasis
,
carcinoma
,
prostatic neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
squamous cell carcinoma of head and neck
,
adenocarcinoma
,
esophageal neoplasms
,
ovarian neoplasms
,
squamous cell carcinoma
,
stomach neoplasms
,
endometrial neoplasms
,
ovarian epithelial carcinoma
,
fallopian tube neoplasms
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
head and neck neoplasms
,
disease progression
,
peritoneal neoplasms
,
squamous cell neoplasms
,
esophageal squamous cell carcinoma
,
breast neoplasms
,
vomiting
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