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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
,
proteinuria
,
neuromuscular diseases
,
membranous glomerulonephritis
,
aspergillosis
,
allergic bronchopulmonary aspergillosis
,
pulmonary aspergillosis
,
limb-girdle muscular dystrophies
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
atrophic muscular disorders
,
chronic renal insufficiency
,
postoperative pain
,
trismus
,
impacted tooth
,
hearing loss
,
otitis media with effusion
,
conductive hearing loss
,
kidney diseases
,
iga glomerulonephritis
,
immunosuppression therapy
,
asthma
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
stomach neoplasms
,
esophageal neoplasms
,
lung neoplasms
,
kidney neoplasms
,
mouth neoplasms
Alfacalcidol
hepatitis c
,
hepatitis
,
hepatitis a
,
fibrosis
,
chronic hepatitis c
,
leukemia
,
myeloid leukemia acute
,
recurrence
Aspartame
fibrosis
,
gastrointestinal diseases
,
cystic fibrosis
,
digestive system diseases
,
pancreatic diseases
,
exocrine pancreatic insufficiency
Bevifimod
hematologic neoplasms
,
biphenotypic leukemia acute
,
b-cell leukemia
,
precursor cell lymphoblastic leukemia-lymphoma
,
stomach neoplasms
,
myelodysplastic syndromes
,
stroke
,
muscle spasticity
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
endometriosis
,
infertility
,
pancreatic ductal carcinoma
,
biliary tract neoplasms
,
esophageal squamous cell carcinoma
,
ovarian neoplasms
,
recurrence
,
b-cell lymphoma
,
b-cell lymphoma marginal zone
,
fallopian tube neoplasms
,
burkitt lymphoma
,
triple negative breast neoplasms
,
squamous cell carcinoma of head and neck
,
multiple myeloma
,
prostatic neoplasms
,
pancreatic neoplasms
,
esophageal neoplasms
,
breast neoplasms
,
reactive arthritis
,
waldenstrom macroglobulinemia
,
lymphoproliferative disorders
,
lymphatic diseases
,
intestinal neoplasms
,
colonic neoplasms
,
rectal neoplasms
,
carcinoma
,
glioblastoma
,
melanoma
,
neuroendocrine carcinoma
,
papillary thyroid cancer
,
adenocarcinoma
,
primary myelofibrosis
,
splenomegaly
,
hairy cell leukemia
,
abdominal hernia
,
ventral hernia
,
urinary bladder neoplasms
,
uterine neoplasms
,
knee osteoarthritis
,
residual neoplasm
,
neuralgia
,
interstitial cystitis
,
neoadjuvant therapy
,
ovarian epithelial carcinoma
,
mantle-cell lymphoma
,
female genital neoplasms
,
peritoneal neoplasms
,
fallopian tube diseases
,
neoplasms
,
non-hodgkin lymphoma
,
crohn disease
,
non-small-cell lung carcinoma
,
b-cell chronic lymphocytic leukemia
,
myeloid leukemia acute
,
colorectal neoplasms
,
lymphoma
,
large b-cell lymphoma diffuse
,
follicular lymphoma
,
lung neoplasms
,
joint diseases
,
musculoskeletal diseases
,
ankylosing spondylitis
,
small cell lung carcinoma
,
macular degeneration
,
multiple sclerosis
,
relapsing-remitting multiple sclerosis
,
inflammatory bowel diseases
,
spondylitis
,
juvenile arthritis
,
neoplasm metastasis
,
skin diseases
,
papulosquamous skin diseases
,
wet macular degeneration
,
hepatocellular carcinoma
,
liver neoplasms
,
infections
,
communicable diseases
,
bacterial infections
,
methicillin-resistant staphylococcus aureus
,
staphylococcal skin infections
,
infectious skin diseases
,
bacterial skin diseases
,
cellulitis
,
disease progression
,
covid-19
,
parkinson disease
,
bronchopulmonary dysplasia
,
respiratory syncytial viruses
,
hepatitis b
,
ulcer
,
pyoderma gangrenosum
,
spondylarthropathies
,
spondylarthritis
,
spinal diseases
,
uveitis
,
intermediate uveitis
,
posterior uveitis
,
precocious puberty
,
migraine disorders
,
retinal diseases
,
retinal degeneration
,
eye diseases
,
choroidal neovascularization
,
leukemia
,
lymphoid leukemia
,
thyroid diseases
,
hypothyroidism
,
rheumatoid arthritis
,
psoriasis
,
arthritis
,
ulcerative colitis
,
psoriatic arthritis
,
hidradenitis suppurativa
,
hepatitis c
,
chronic hepatitis c
,
axial spondyloarthritis
,
cardiovascular diseases
,
connective tissue diseases
,
seizures
,
panuveitis
,
hiv infections
,
drug monitoring
,
coronary artery disease
,
myocardial ischemia
,
inflammation
,
atherosclerosis
,
hyperhidrosis
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
chronic renal insufficiency
,
gout
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
myelodysplastic syndromes
,
lymphoid leukemia
,
thoracic neoplasms
,
neurologic manifestations
,
pharmacokinetics
,
soft tissue neoplasms
,
precursor cell lymphoblastic leukemia-lymphoma
,
respiratory tract neoplasms
,
bronchogenic carcinoma
,
endocrine gland neoplasms
,
breast diseases
,
bronchial neoplasms
,
intraductal carcinoma noninfiltrating
,
laryngeal diseases
,
myoepithelioma
,
astrocytoma
,
oligodendroglioma
,
oral administration
,
pancreatic neoplasms
,
lymphoma
,
sarcoma
,
brain neoplasms
,
castration-resistant prostatic neoplasms
,
large b-cell lymphoma diffuse
,
non-hodgkin lymphoma
,
b-cell lymphoma
,
follicular lymphoma
,
myeloid leukemia acute
,
kidney neoplasms
,
leukemia
,
t-cell lymphoma
,
colonic neoplasms
,
neuroendocrine tumors
,
pancreatic ductal carcinoma
,
uveal melanoma
,
uveal neoplasms
,
glioma
,
invasive hydatidiform mole
,
neuroendocrine carcinoma
,
t-cell lymphoma peripheral
,
thyroid neoplasms
,
myeloid leukemia
,
anus neoplasms
,
cutaneous malignant melanoma
,
gastrointestinal neoplasms
,
mouth neoplasms
,
oropharyngeal neoplasms
,
small cell carcinoma
,
urethral neoplasms
,
uterine neoplasms
,
b-cell lymphoma marginal zone
,
neoplasms by histologic type
,
female genital neoplasms
,
hypersensitivity
,
thymoma
,
epstein-barr virus infections
,
microsatellite repeats
,
hypopharyngeal neoplasms
,
salivary gland neoplasms
,
endometrioid carcinoma
,
vulvar neoplasms
,
mantle-cell lymphoma
,
waldenstrom macroglobulinemia
,
non-muscle invasive bladder neoplasms
,
penile neoplasms
,
liposarcoma
,
b-cell chronic lymphocytic leukemia
,
t-cell lymphoma cutaneous
,
ureteral neoplasms
,
osteosarcoma
,
gliosarcoma
,
carcinosarcoma
,
thymus neoplasms
,
burkitt lymphoma
,
rectal neoplasms
,
laryngeal neoplasms
,
disease
,
lung diseases
,
adenoid cystic carcinoma
,
meningioma
,
serous cystadenocarcinoma
,
glandular and epithelial neoplasms
,
male breast neoplasms
,
leiomyosarcoma
,
nasopharyngeal carcinoma
,
adenocarcinoma of lung
,
oncogenes
,
neurofibrosarcoma
,
nerve sheath neoplasms
,
mycosis fungoides
,
syndrome
,
interdigitating dendritic cell sarcoma
,
central nervous system neoplasms
,
germ cell and embryonal neoplasms
,
digestive system neoplasms
,
basal cell carcinoma
,
reactive arthritis
,
adenosquamous carcinoma
,
gestational trophoblastic disease
,
alveolar soft part sarcoma
,
biphenotypic leukemia acute
,
local neoplasm recurrence
,
sclerosis
,
chordoma
,
meningeal carcinomatosis
,
papillomaviridae
,
immunoblastic lymphadenopathy
,
enteropathy-associated t-cell lymphoma
,
vaccinia
,
carcinoma in situ
,
cervical intraepithelial neoplasia
,
uterine cervical dysplasia
,
squamous intraepithelial lesions of the cervix
,
disease progression
,
central nervous system diseases
,
extranodal nk-t-cell lymphoma
,
neoplasms by site
,
x-linked bulbo-spinal atrophy
,
malignant fibrous histiocytoma
,
virus diseases
,
viremia
,
unknown primary neoplasms
,
synovial sarcoma
,
point mutation
,
oral leukoplakia
,
leukoplakia
,
hematologic diseases
,
bone marrow diseases
,
mycoses
,
sezary syndrome
,
immune system diseases
,
lymphoproliferative disorders
,
lymphatic diseases
,
immunoproliferative disorders
,
endoscopy
,
colonic diseases
,
histiocytic sarcoma
,
follicular dendritic cell sarcoma
,
pelvic neoplasms
,
environmental biomarkers
,
inflammatory breast neoplasms
,
meningeal neoplasms
,
intestinal neoplasms
,
nervous system neoplasms
,
kidney diseases
,
nervous system diseases
,
nerve tissue neoplasms
,
neuroepithelial neoplasms
,
neuroectodermal tumors
,
anaplastic thyroid carcinoma
,
thyroid diseases
,
adrenocortical carcinoma
,
carcinoid tumor
,
parotid neoplasms
,
mediastinal neoplasms
,
testicular neoplasms
,
male genital neoplasms
,
gastrointestinal stromal tumors
,
hairy cell leukemia
,
spinal cord neoplasms
,
acinar cell carcinoma
,
adenoviridae infections
,
hypoxia
,
hereditary breast and ovarian cancer syndrome
,
basal cell neoplasms
,
urogenital neoplasms
,
male urogenital diseases
,
hemangiosarcoma
,
perivascular epithelioid cell neoplasms
,
carcinoma
,
breast neoplasms
,
lung neoplasms
,
colorectal neoplasms
,
triple negative breast neoplasms
,
renal cell carcinoma
,
ovarian neoplasms
,
squamous cell carcinoma
,
neoplasm metastasis
,
head and neck neoplasms
,
urinary bladder neoplasms
,
adenocarcinoma
,
prostatic neoplasms
,
liver neoplasms
,
recurrence
,
esophageal neoplasms
,
endometrial neoplasms
,
hepatocellular carcinoma
,
transitional cell carcinoma
,
hodgkin disease
,
urologic neoplasms
,
small cell lung carcinoma
,
fallopian tube neoplasms
,
glioblastoma
,
multiple myeloma
,
squamous cell neoplasms
,
ovarian epithelial carcinoma
,
mesothelioma
,
esophageal squamous cell carcinoma
,
cholangiocarcinoma
,
plasma cell neoplasms
,
skin neoplasms
,
malignant mesothelioma
,
peritoneal neoplasms
,
merkel cell carcinoma
,
microsatellite instability
,
nasopharyngeal neoplasms
,
bile duct neoplasms
,
gallbladder neoplasms
,
hematologic neoplasms
,
non-small-cell lung carcinoma
,
neoplasms
,
melanoma
,
squamous cell carcinoma of head and neck
,
stomach neoplasms
,
uterine cervical neoplasms
,
biliary tract neoplasms
Solumedrol
macular edema
,
retinal diseases
,
diabetic retinopathy
,
uveitis
,
anterior uveitis
,
iridocyclitis
,
inflammation
,
cataract
,
edema
,
corneal edema
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