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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
,
limb-girdle muscular dystrophies
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
myelodysplastic syndromes
,
pharmacokinetics
,
thoracic neoplasms
,
invasive hydatidiform mole
,
lymphoma
,
pancreatic neoplasms
,
b-cell lymphoma
,
sarcoma
,
large b-cell lymphoma diffuse
,
glioblastoma
,
castration-resistant prostatic neoplasms
,
brain neoplasms
,
non-hodgkin lymphoma
,
mesothelioma
,
microsatellite instability
,
colonic neoplasms
,
follicular lymphoma
,
t-cell lymphoma
,
neuroendocrine tumors
,
hypersensitivity
,
epstein-barr virus infections
,
myeloid leukemia acute
,
mouth neoplasms
,
oropharyngeal neoplasms
,
meningioma
,
pancreatic ductal carcinoma
,
adenocarcinoma of lung
,
oncogenes
,
t-cell lymphoma peripheral
,
syndrome
,
urethral neoplasms
,
ureteral neoplasms
,
glioma
,
local neoplasm recurrence
,
rectal neoplasms
,
laryngeal neoplasms
,
hypopharyngeal neoplasms
,
chordoma
,
leiomyosarcoma
,
liposarcoma
,
malignant fibrous histiocytoma
,
nasopharyngeal carcinoma
,
virus diseases
,
viremia
,
unknown primary neoplasms
,
leukemia
,
hematologic diseases
,
b-cell chronic lymphocytic leukemia
,
bone marrow diseases
,
endometrioid carcinoma
,
mycoses
,
sezary syndrome
,
mycosis fungoides
,
t-cell lymphoma cutaneous
,
colonic diseases
,
histiocytic sarcoma
,
follicular dendritic cell sarcoma
,
interdigitating dendritic cell sarcoma
,
pelvic neoplasms
,
environmental biomarkers
,
inflammatory breast neoplasms
,
intestinal neoplasms
,
kidney diseases
,
kidney neoplasms
,
gliosarcoma
,
disease
,
nervous system diseases
,
neoplasms by histologic type
,
nerve tissue neoplasms
,
neuroepithelial neoplasms
,
neuroectodermal tumors
,
salivary gland neoplasms
,
thyroid neoplasms
,
anus neoplasms
,
carcinoid tumor
,
vulvar neoplasms
,
parotid neoplasms
,
reactive arthritis
,
gastrointestinal stromal tumors
,
osteosarcoma
,
central nervous system neoplasms
,
spinal cord neoplasms
,
adenoviridae infections
,
gastrointestinal neoplasms
,
digestive system neoplasms
,
hypoxia
,
uterine neoplasms
,
carcinosarcoma
,
mullerian mixed tumor
,
basal cell carcinoma
,
basal cell neoplasms
,
astrocytoma
,
male breast neoplasms
,
uveal neoplasms
,
uveal melanoma
,
neoplasms
,
carcinoma
,
lung neoplasms
,
colorectal neoplasms
,
triple negative breast neoplasms
,
breast neoplasms
,
renal cell carcinoma
,
squamous cell carcinoma
,
head and neck neoplasms
,
ovarian neoplasms
,
neoplasm metastasis
,
urinary bladder neoplasms
,
liver neoplasms
,
stomach neoplasms
,
hepatocellular carcinoma
,
adenocarcinoma
,
prostatic neoplasms
,
endometrial neoplasms
,
esophageal neoplasms
,
hodgkin disease
,
recurrence
,
small cell lung carcinoma
,
uterine cervical neoplasms
,
ovarian epithelial carcinoma
,
squamous cell neoplasms
,
transitional cell carcinoma
,
multiple myeloma
,
urologic neoplasms
,
esophageal squamous cell carcinoma
,
fallopian tube neoplasms
,
biliary tract neoplasms
,
skin neoplasms
,
cholangiocarcinoma
,
cutaneous malignant melanoma
,
nasopharyngeal neoplasms
,
plasma cell neoplasms
,
bile duct neoplasms
,
merkel cell carcinoma
,
gallbladder neoplasms
,
malignant mesothelioma
,
peritoneal neoplasms
,
hematologic neoplasms
,
non-muscle invasive bladder neoplasms
,
non-small-cell lung carcinoma
,
melanoma
,
squamous cell carcinoma of head and neck
Alfacalcidol
friedreich ataxia
Aspartame
leukemia
,
myeloid leukemia
,
ulcerative colitis
,
colitis
,
ulcer
,
rheumatoid arthritis
,
myeloid leukemia acute
,
atopic dermatitis
,
hidradenitis suppurativa
,
eczema
,
dermatitis
,
crohn disease
,
pathologic constriction
,
inflammatory bowel diseases
,
intestinal diseases
Bevifimod
adenocarcinoma
,
prostatic neoplasms
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
healthy volunteers/patients
,
melanoma
,
urinary tract infections
,
gram-negative bacterial infections
,
neoplasms
,
non-small-cell lung carcinoma
,
triple negative breast neoplasms
,
neoplasm metastasis
,
soft tissue infections
,
genomic instability
,
bacterial pneumonia
,
pancreatic neoplasms
,
urinary bladder neoplasms
,
cholangiocarcinoma
,
ovarian neoplasms
,
brain neoplasms
,
stomach neoplasms
,
inflammatory breast neoplasms
,
patient preference
,
breast neoplasms
,
pneumonia
,
cystic fibrosis
,
pseudomonas infections
,
pseudomonas aeruginosa
,
hiv infections
,
hiv seropositivity
,
neurosyphilis
Mk3475
carcinoma
,
renal cell carcinoma
,
colorectal neoplasms
,
lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
breast neoplasms
,
nasopharyngeal carcinoma
,
squamous cell carcinoma of head and neck
,
anus neoplasms
,
squamous cell carcinoma
,
malignant mesothelioma
,
thymoma
,
microsatellite instability
,
ovarian epithelial carcinoma
,
nasopharyngeal neoplasms
,
mesothelioma
,
thymus neoplasms
,
cutaneous malignant melanoma
,
melanoma
,
neoplasms
,
lung neoplasms
,
triple negative breast neoplasms
,
non-small-cell lung carcinoma
,
neoplasm metastasis
Solumedrol
graft rejection
,
organ transplantation
,
psychology rejection
,
melanoma
,
leukemia
,
myelodysplastic syndromes
,
myeloid leukemia acute
,
myeloid leukemia
,
preleukemia
,
syndrome
,
renal cell carcinoma
,
lung neoplasms
,
synovitis
,
lymphoma
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
immunoglobulin g4-related disease
,
systemic lupus erythematosus
,
polymyalgia rheumatica
,
giant cell arteritis
,
kidney transplantation
,
epstein-barr virus infections
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