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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
,
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
,
kidney diseases
,
iga glomerulonephritis
,
immunosuppression therapy
,
asthma
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
head and neck neoplasms
,
squamous cell carcinoma of head and neck
Alfacalcidol
renal cell carcinoma
,
neoplasms
,
uterine cervical neoplasms
,
anemia
,
chronic hepatitis d
,
hepatitis d
,
rheumatoid arthritis
,
hepatitis
,
hepatitis a
,
hepatitis c
,
chronic hepatitis c
,
chronic hepatitis
Aspartame
phenylketonurias
Bevifimod
hemolytic-uremic syndrome
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
uveitis
,
precursor cell lymphoblastic leukemia-lymphoma
,
neoplasms
,
respiratory insufficiency
,
pneumonia
,
bronchiolitis obliterans syndrome
,
giant cell arteritis
,
polymyalgia rheumatica
,
lupus nephritis
,
multiple myeloma
,
breast neoplasms
,
stomatitis
,
oral ulcer
,
animal mammary neoplasms
,
ornithine carbamoyltransferase deficiency disease
,
covid-19
,
arteritis
,
tendinopathy
,
sarcoidosis
,
pulmonary sarcoidosis
,
leukemia
,
lymphoma
,
b-cell chronic lymphocytic leukemia
,
hypersensitivity
,
myositis
,
healthy volunteers/patients
,
prurigo
,
proteinuria
,
systemic lupus erythematosus
,
prostatic neoplasms
,
urogenital neoplasms
,
male genital neoplasms
,
hematopoietic stem cell transplantation
,
vasculitis
,
granulomatosis with polyangiitis
,
anti-neutrophil cytoplasmic antibody-associated vasculitis
,
microscopic polyangiitis
,
obstructive lung diseases
,
lung diseases
,
thrombocytopenic purpura idiopathic
,
psychology rejection
,
graft rejection
,
follicular lymphoma
,
hemorrhage
,
thrombocytopenia
,
heart diseases
,
nephrotic syndrome
,
syndrome
,
nephrosis
,
osteoporosis
,
recurrence
,
kidney transplantation
,
asthma
,
chronic obstructive pulmonary disease
,
graft vs host disease
,
liver transplantation
,
atopic dermatitis
,
eczema
,
dermatitis
,
renal insufficiency
,
chronic kidney failure
,
kidney diseases
,
infections
,
cytomegalovirus infections
,
communicable diseases
,
heart transplantation
,
multiple sclerosis
,
sclerosis
,
chronic renal insufficiency
,
renal dialysis
,
renal replacement therapy
,
drug-related side effects and adverse reactions
,
fatigue
,
relapsing-remitting multiple sclerosis
,
cystic fibrosis
,
allergic bronchopulmonary aspergillosis
,
transplantation
,
spinal muscular atrophy
,
end stage liver disease
,
emergencies
,
disease progression
,
psoriasis
Lactams
hiv
,
cystic fibrosis
Mk3475
neoplasms
,
breast neoplasms
,
melanoma
,
carcinoma
,
pancreatic neoplasms
,
liver neoplasms
,
renal cell carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
head and neck neoplasms
,
sarcoma
,
lymphoma
,
bile duct neoplasms
,
colonic neoplasms
,
squamous cell carcinoma
,
squamous cell neoplasms
,
chordoma
,
endometrial neoplasms
,
hepatocellular carcinoma
,
stomach neoplasms
,
mesothelioma
,
triple negative breast neoplasms
,
colorectal neoplasms
,
pancreatic ductal carcinoma
,
non-small-cell lung carcinoma
,
lung neoplasms
Solumedrol
leukemia
,
giant cell arteritis
,
polymyalgia rheumatica
,
precursor cell lymphoblastic leukemia-lymphoma
,
arteritis
,
lymphoma
,
b-cell chronic lymphocytic leukemia
,
breast neoplasms
,
stomatitis
,
oral ulcer
,
animal mammary neoplasms
,
lymphoid leukemia
,
myositis
,
graft vs host disease
,
bronchiolitis obliterans syndrome
,
lupus nephritis
,
carcinoma
,
peritoneal neoplasms
,
intestinal obstruction
,
intermediate uveitis
,
recurrence
,
follicular lymphoma
,
prostatic neoplasms
,
kidney transplantation
,
liver transplantation
,
chronic kidney failure
,
neoplasms
,
infections
,
healthy volunteers/patients
,
chronic renal insufficiency
,
kidney diseases
,
renal dialysis
,
renal replacement therapy
,
renal insufficiency
,
liver cirrhosis
,
drug-related side effects and adverse reactions
,
cytomegalovirus infections
,
communicable diseases
,
multiple sclerosis
,
fatigue
,
relapsing-remitting multiple sclerosis
,
sclerosis
,
heart transplantation
,
uveitis
,
cataract
,
inflammation
,
cataract extraction
,
capsule opacification
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