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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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Therapeutic Areas:
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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
,
friedreich ataxia
,
cerebellar ataxia
,
mitochondrial diseases
,
syndrome
,
leigh disease
,
brain diseases
,
epilepsy
,
diffuse cerebral sclerosis of schilder
,
drug resistant epilepsy
,
melas syndrome
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
infections
,
chronic hepatitis d
,
hepatitis b
,
chronic hepatitis b
,
communicable diseases
,
hepatitis d
,
hepatitis
,
chronic hepatitis
,
hepatitis a
,
neoplasms
,
carcinoma
,
hepatocellular carcinoma
,
liver neoplasms
Aspartame
tuberculosis
,
neoplasms
,
leukemia
,
myeloid leukemia acute
,
myeloid leukemia
,
deficiency diseases
,
paraproteinemias
,
hiv infections
,
precursor cell lymphoblastic leukemia-lymphoma
,
lymphoid leukemia
,
breast neoplasms
,
immune system diseases
,
hematologic neoplasms
,
type 2 diabetes mellitus
,
uveitis
,
metabolic diseases
,
disease
,
lipidoses
,
immunoglobulin g4-related disease
,
malaria
,
falciparum malaria
,
whooping cough
,
tetanus
,
diphtheria
,
tetany
,
polycystic kidney diseases
,
autosomal dominant polycystic kidney
,
arthrogryposis
,
renal insufficiency
,
hematologic diseases
,
blood protein disorders
,
neoplasms by histologic type
,
prostatic neoplasms
,
circulating neoplastic cells
,
heart transplantation
,
non-small-cell lung carcinoma
,
lung neoplasms
,
carcinoma
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
esophageal neoplasms
,
adenocarcinoma
,
neoplasm metastasis
,
anemia
,
aplastic anemia
,
multiple myeloma
,
plasma cell neoplasms
,
hypercholesterolemia
,
hyperlipoproteinemia type ii
,
meningococcal infections
,
hemolytic anemia autoimmune
,
meningitis
,
meningococcal meningitis
,
stomach neoplasms
,
hemophilia a
,
kidney diseases
,
gaucher disease
,
smoldering multiple myeloma
,
hemophilia b
,
chronic renal insufficiency
,
hyperphosphatemia
,
multiple sclerosis
,
sclerosis
,
homozygous familial hypercholesterolemia
,
pharyngitis
,
tonsillitis
,
healthy volunteers/patients
,
asthma
,
glycogen storage disease type ii
,
glycogen storage disease
,
atopic dermatitis
,
dermatitis
,
lymphoma
,
eczema
,
diabetes mellitus
,
hyperglycemia
,
glycogen storage disease type iii
,
non-hodgkin lymphoma
,
osteoporosis
,
postmenopausal osteoporosis
,
lymphoproliferative disorders
Bevifimod
pancreatic neoplasms
,
inflammatory bowel diseases
,
waldenstrom macroglobulinemia
,
myeloid leukemia acute
,
myelodysplastic syndromes
,
renal insufficiency
,
brain neoplasms
,
glandular and epithelial neoplasms
,
myoepithelioma
,
delayed graft function
,
hypersensitivity
,
venous thromboembolism
,
thromboembolism
,
pharmacokinetics
,
gliosarcoma
,
central nervous system diseases
,
brain diseases
,
hiv infections
,
endometrial neoplasms
,
carcinosarcoma
,
mullerian mixed tumor
,
ovarian neoplasms
,
neoplasms
,
melanoma
,
breast neoplasms
,
rheumatoid arthritis
,
adenocarcinoma
,
b-cell chronic lymphocytic leukemia
,
neoplasm metastasis
,
glioblastoma
,
non-hodgkin lymphoma
,
blast crisis
,
colorectal neoplasms
,
small cell lung carcinoma
,
ulcerative colitis
,
colitis
,
ulcer
,
lymphoma
,
triple negative breast neoplasms
,
autoimmune diseases
,
crohn disease
,
kidney neoplasms
,
female genital neoplasms
,
biliary tract neoplasms
,
neuroendocrine tumors
,
chordoma
,
kidney transplantation
,
glioma
,
diffuse intrinsic pontine glioma
,
nephrotic syndrome
,
focal segmental glomerulosclerosis
,
nephrosis
,
lipoid nephrosis
,
discoid lupus erythematosus
,
lupus nephritis
,
nephritis
,
enteritis
,
diabetic nephropathies
,
kidney diseases
,
atopic dermatitis
,
eczema
,
dermatitis
,
alopecia areata
,
alopecia
,
hematologic neoplasms
,
gastrointestinal neoplasms
,
esophageal neoplasms
,
pancreatic ductal carcinoma
,
hidradenitis suppurativa
,
hidradenitis
,
polycythemia vera
,
polycythemia
,
lichen planus
,
central nervous system neoplasms
,
nervous system neoplasms
,
sarcoma
,
bone neoplasms
,
gastrointestinal stromal tumors
,
chondrosarcoma
,
alveolar soft part sarcoma
,
solitary fibrous tumors
,
ewing sarcoma
,
rhabdomyosarcoma
,
primitive neuroectodermal tumors peripheral
,
neurofibrosarcoma
,
nerve sheath neoplasms
,
giant cell tumor of bone
,
giant cell tumors
,
hemangiopericytoma
,
adenocarcinoma of lung
,
merkel cell carcinoma
,
castration-resistant prostatic neoplasms
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
carcinoma
,
non-small-cell lung carcinoma
,
lung neoplasms
,
renal cell carcinoma
,
lymphoid leukemia
,
multiple myeloma
,
philadelphia chromosome
,
arthritis
,
prostatic neoplasms
,
plasma cell neoplasms
,
precursor cell lymphoblastic leukemia-lymphoma
,
psoriatic arthritis
,
hepatocellular carcinoma
,
liver neoplasms
,
syndrome
,
head and neck neoplasms
,
squamous cell carcinoma of head and neck
,
recurrence
,
myeloid leukemia accelerated phase
,
sjogren's syndrome
,
squamous cell carcinoma
,
rheumatic diseases
,
juvenile arthritis
,
leukemia
,
bcr-abl positive chronic myelogenous leukemia
,
myeloid leukemia
,
psoriasis
,
healthy volunteers/patients
,
systemic lupus erythematosus
,
myeloid leukemia chronic-phase
,
cutaneous lupus erythematosus
,
coronary artery disease
,
pleural effusion
,
liver diseases
,
necrosis
,
cardiomyopathies
,
dilated cardiomyopathy
,
alzheimer disease
,
progressive supranuclear palsy
,
paralysis
,
preleukemia
,
urinary bladder neoplasms
,
transitional cell carcinoma
Ubidecarenone
glaucoma
,
open-angle glaucoma
,
alzheimer disease
,
aging
,
myocardial infarction
,
hypoxia
,
oligospermia
,
renal cell carcinoma
,
ovarian neoplasms
,
urinary bladder neoplasms
,
fallopian tube neoplasms
,
melanoma
,
ovarian epithelial carcinoma
,
gliosarcoma
,
astrocytoma
,
barrett esophagus
,
chronic progressive multiple sclerosis
,
vitamin d deficiency
,
hypersensitivity
,
oral lichen planus
,
lichen planus
,
epidermolysis bullosa
,
skin care
,
brain ischemia
,
absorption
,
burns
,
sleep apnea syndromes
,
vestibular diseases
,
crohn disease
,
peripheral nervous system diseases
,
sclerosis
,
covid-19
,
keratoconjunctivitis sicca
,
dry eye syndromes
,
sarcopenia
,
retinal diseases
,
diabetic retinopathy
,
malnutrition
,
stomach neoplasms
,
pancreatic neoplasms
,
leukemia
,
brain diseases
,
multiple sclerosis
,
heart arrest
,
amyotrophic lateral sclerosis
,
motor neuron disease
,
depression
,
bipolar disorder
,
post-acute covid-19 syndrome
,
glioblastoma
,
adenocarcinoma
,
non-small-cell lung carcinoma
,
precursor cell lymphoblastic leukemia-lymphoma
,
second primary neoplasms
,
type 2 diabetes mellitus
,
chromosome deletion
,
autistic disorder
,
autism spectrum disorder
,
type 1 diabetes mellitus
,
diastolic heart failure
,
frailty
,
cystic fibrosis
,
fibrosis
,
renal insufficiency
,
squamous cell carcinoma
,
squamous cell neoplasms
,
paralysis
,
recurrence
,
burkitt lymphoma
,
neutropenia
,
thrombocytopenia
,
acidosis
,
lactic acidosis
,
melas syndrome
,
mantle-cell lymphoma
,
lymphoid leukemia
,
aids-related lymphoma
,
spinal cord injuries
,
prader-willi syndrome
,
post-cardiac arrest syndrome
,
tuberculosis
,
pulmonary tuberculosis
,
rectal neoplasms
,
cri-du-chat syndrome
,
macrocytic anemia
,
hepatitis b
,
chronic hepatitis b
,
hepatitis
,
chronic hepatitis
,
hepatitis a
,
atopic dermatitis
,
eczema
,
dermatitis
,
hearing loss
,
noise-induced hearing loss
,
deafness
,
leigh disease
,
sudden death cardiac
,
down syndrome
,
basal cell carcinoma
,
nerve compression syndromes
,
tooth diseases
,
charcot-marie-tooth disease
,
hereditary sensory and motor neuropathy
,
mood disorders
,
bipolar and related disorders
,
cysts
,
jaw cysts
,
metabolic diseases
,
disease
,
glycogen storage disease type ii
,
lysosomal storage diseases
,
glycogen storage disease
,
liver diseases
,
pervasive child development disorders
,
systemic lupus erythematosus
,
syndrome
,
fatigue
,
mitochondrial diseases
,
healthy volunteers/patients
,
colorectal neoplasms
,
parkinson disease
,
chronic fatigue syndrome
,
lymphoma
,
ataxia
,
breast neoplasms
,
coronary artery disease
,
heart failure
,
neoplasms
,
carcinoma
,
polycystic ovary syndrome
,
prostatic neoplasms
,
cerebellar ataxia
,
huntington disease
,
myocardial ischemia
,
lung neoplasms
,
friedreich ataxia
,
osteoporosis
,
obesity
,
progressive supranuclear palsy
,
duchenne muscular dystrophy
,
muscular dystrophies
,
myelodysplastic syndromes
,
coronary disease
,
drug-related side effects and adverse reactions
,
non-alcoholic fatty liver disease
,
fatty liver
,
cognitive dysfunction
,
cholangiocarcinoma
,
liver neoplasms
,
neoplasm metastasis
,
headache
,
biliary tract neoplasms
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
dyslipidemias
,
myositis
,
persian gulf syndrome
,
preleukemia
,
hyperlipidemias
,
hyperlipoproteinemias
,
schizophrenia
,
pain
,
gastrointestinal neoplasms
,
neuroendocrine tumors
,
carcinoid tumor
,
paraneoplastic syndromes
,
islet cell adenoma
,
malignant carcinoid syndrome
,
rem sleep behavior disorder
,
mental disorders
,
headache disorders
,
nervous system diseases
,
hepatocellular carcinoma
,
gallbladder neoplasms
,
ampulla of vater cancer
,
pathologic dilatation
,
keratoconus
,
corneal diseases
,
diarrhea
,
multiple organ failure
,
apraxias
,
cogan syndrome
,
limb-girdle muscular dystrophies
,
muscle cramp
,
coronary artery bypass
,
cardiac valve annuloplasty
,
heart diseases
,
coronary stenosis
,
pre-eclampsia
,
juvenile arthritis
,
arthritis
,
overweight
,
weight loss
,
myotonic dystrophy
,
tic disorders
,
infertility
,
cardiovascular diseases
,
inflammation
,
male infertility
,
hypercholesterolemia
,
migraine disorders
,
diabetes mellitus
,
metabolic syndrome
,
oxidative stress
,
kidney diseases
,
myalgia
,
atherosclerosis
,
sepsis
,
septic shock
,
toxemia
,
chronic kidney failure
,
insulin resistance
,
shock
,
renal dialysis
,
chronic pain
,
hypertriglyceridemia
,
epilepsy
,
chronic renal insufficiency
,
critical illness
,
stroke
,
infections
,
opioid-related disorders
,
morphine dependence
,
bone fractures
,
gastritis
,
semen analysis
,
iron overload
,
muscle weakness
,
cardiomyopathies
,
dilated cardiomyopathy
,
severe acute respiratory syndrome-related coronavirus
,
severe acute respiratory syndrome
,
myocarditis
,
virus diseases
,
dry socket
,
temporomandibular joint disorders
,
joint diseases
,
temporomandibular joint dysfunction syndrome
,
acute coronary syndrome
,
attitudes health knowledge practice
,
postoperative pain
,
periodontal diseases
,
macular edema
,
vomiting
,
melanosis
,
hypertension
,
aneuploidy
,
muscular diseases
,
depressive disorder
,
edema
,
myofascial pain syndromes
,
fibromyalgia
,
hemophilia b
,
medication adherence
,
chromosome disorders
,
anemia
,
fetal growth retardation
,
premature birth
,
iron deficiencies
,
vitamin b 12 deficiency
,
morbid obesity
,
dermatomyositis
,
female infertility
,
micronutrients
,
cardiovascular abnormalities
,
ascorbic acid deficiency
,
ovarian reserve
,
vascular remodeling
,
macular degeneration
,
wet macular degeneration
,
metabolism
,
post-concussion syndrome
,
gestational diabetes
,
pregnancy outcome
,
gingival recession
,
acute kidney injury
,
intellectual disability
,
hepatic encephalopathy
,
psychotic disorders
,
health status indicators
,
aphthous stomatitis
,
ulcer
,
tinnitus
,
hyperkinesis
,
attention deficit disorder with hyperactivity
,
thrombosis
,
mitochondrial genome
,
reproductive physiological phenomena
,
inborn genetic diseases
,
pulmonary arterial hypertension
,
familial primary pulmonary hypertension
,
spontaneous abortion
,
pregnancy
,
kidney transplantation
,
ischemic preconditioning
,
exercise
,
respiratory tract infections
,
encephalomyelitis
,
dietary supplements
,
healthy diet
,
only child
,
dental implants
,
hyperglycemia
,
pregnancy rate
,
chronic periodontitis
,
periodontitis
,
oral hygiene
,
asthenia
,
physical fitness
,
liver cirrhosis
,
kwashiorkor
,
st elevation myocardial infarction
,
infarction
,
communicable diseases
,
varicocele
,
alveolar bone grafting
,
atrial fibrillation
,
hyperpigmentation
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
multiple myeloma
,
plasma cell neoplasms
,
melanoma
,
heart transplantation
,
graft rejection
,
brain neoplasms
,
renal cell carcinoma
,
lung neoplasms
,
diarrhea
,
immunoglobulin g4-related disease
,
neoplasms
,
covid-19
,
lupus nephritis
,
nephritis
,
systemic lupus erythematosus
,
vasculitis
,
granulomatosis with polyangiitis
,
anti-neutrophil cytoplasmic antibody-associated vasculitis
,
systemic vasculitis
,
kidney transplantation
,
renal insufficiency
,
epstein-barr virus infections
Mk3475
lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
prostatic neoplasms
,
castration-resistant prostatic neoplasms
,
myeloid leukemia acute
,
myeloid leukemia
,
small cell lung carcinoma
,
head and neck neoplasms
,
squamous cell carcinoma of head and neck
,
squamous cell carcinoma
,
squamous cell neoplasms
,
thoracic neoplasms
,
neoplasms
,
leukemia
,
sarcoma
,
carcinoma
,
hemangiosarcoma
,
hepatocellular carcinoma
,
liver neoplasms
,
precursor cell lymphoblastic leukemia-lymphoma
,
biphenotypic leukemia acute
,
lymphoid leukemia
,
renal cell carcinoma
,
kidney neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
melanoma
Solumedrol
graft vs host disease
Docetaxol
salivary gland neoplasms
,
thyroid neoplasms
,
melanoma
,
neoplasms
,
esophageal neoplasms
,
stomach neoplasms
,
prostatic neoplasms
,
head and neck neoplasms
,
adenocarcinoma
,
carcinoma
,
neoplasm metastasis
,
sarcoma
,
squamous cell carcinoma
,
brain neoplasms
,
recurrence
,
local neoplasm recurrence
,
diarrhea
,
leiomyosarcoma
,
hemangiosarcoma
,
histiocytoma
,
malignant fibrous histiocytoma
,
squamous cell neoplasms
,
urologic neoplasms
,
transitional cell carcinoma
,
ovarian neoplasms
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
breast neoplasms
,
lung neoplasms
,
non-small-cell lung carcinoma
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