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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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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
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
neuromuscular diseases
,
atrophic muscular disorders
,
fibrosis
,
cystic fibrosis
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
healthy volunteers/patients
,
second primary neoplasms
,
communicable diseases
,
hiv infections
,
synovial sarcoma
,
myxoid liposarcoma
,
b-cell leukemia
,
chronic hepatitis d
,
hepatitis d
,
alveolar soft part sarcoma
,
hepatitis c
,
thymus neoplasms
,
bone neoplasms
,
large cell carcinoma
,
thrombosis
,
liver transplantation
,
urethral neoplasms
,
ureteral neoplasms
,
pelvic neoplasms
,
meningeal neoplasms
,
meningeal carcinomatosis
,
t-cell lymphoma cutaneous
,
urogenital neoplasms
,
mycoses
,
stomatitis
,
mycosis fungoides
,
vesicular stomatitis
,
local neoplasm recurrence
,
sarcoma
,
neoadjuvant therapy
,
castration-resistant prostatic neoplasms
,
pancreatic ductal carcinoma
,
mesothelioma
,
b-cell lymphoma
,
glioblastoma
,
adenocarcinoma of lung
,
thyroid neoplasms
,
leukemia
,
microsatellite instability
,
multiple myeloma
,
parkinson disease
,
gastrointestinal neoplasms
,
plasma cell neoplasms
,
cutaneous malignant melanoma
,
myeloid leukemia acute
,
endometrioid carcinoma
,
neuroendocrine tumors
,
osteosarcoma
,
digestive system neoplasms
,
colonic neoplasms
,
myeloid leukemia
,
drug-related side effects and adverse reactions
,
oropharyngeal neoplasms
,
chordoma
,
residual neoplasm
,
clear cell adenocarcinoma
,
thyroid diseases
,
follicular lymphoma
,
glioma
,
non-hodgkin lymphoma
,
liposarcoma
,
extranodal nk-t-cell lymphoma
,
myelodysplastic syndromes
,
adenomyoepithelioma
,
female genital neoplasms
,
basal cell carcinoma
,
basal cell neoplasms
,
hematologic neoplasms
,
salivary gland neoplasms
,
mouth neoplasms
,
malignant mesothelioma
,
precursor cell lymphoblastic leukemia-lymphoma
,
neuroendocrine carcinoma
,
pneumonia
,
mucinous adenocarcinoma
,
cystadenocarcinoma
,
serous cystadenocarcinoma
,
gastrointestinal stromal tumors
,
adenoid cystic carcinoma
,
adrenocortical carcinoma
,
vulvar neoplasms
,
large-cell lymphoma anaplastic
,
peritoneal neoplasms
,
malignant fibrous histiocytoma
,
astrocytoma
,
lung diseases
,
acquired immunodeficiency syndrome
,
hyperthermia
,
b-cell chronic lymphocytic leukemia
,
mantle-cell lymphoma
,
ascites
,
therapeutic chemoembolization
,
uterine neoplasms
,
bronchogenic carcinoma
,
small cell carcinoma
,
syndrome
,
lymphoid leukemia
,
tertiary lymphoid structures
,
organ preservation
,
anaplastic thyroid carcinoma
,
bcr-abl positive chronic myelogenous leukemia
,
myeloid leukemia chronic-phase
,
unknown primary neoplasms
,
pheochromocytoma
,
mucinous cystadenocarcinoma
,
germ cell and embryonal neoplasms
,
teratoma
,
paraganglioma
,
gallbladder neoplasms
,
hemangiosarcoma
,
bronchiolo-alveolar adenocarcinoma
,
islet cell carcinoma
,
choriocarcinoma
,
parathyroid neoplasms
,
rare diseases
,
sex cord-gonadal stromal tumors
,
neurofibrosarcoma
,
mesothelial neoplasms
,
papillary adenocarcinoma
,
acinar cell carcinoma
,
perivascular epithelioid cell neoplasms
,
trophoblastic neoplasms
,
paranasal sinus neoplasms
,
seminoma
,
nerve sheath neoplasms
,
giant cell carcinoma
,
fibroma
,
desmoid tumors
,
pseudomyxoma peritonei
,
extramammary paget disease
,
spinal neoplasms
,
tracheal neoplasms
,
prostatic diseases
,
fibrosarcoma
,
bronchopulmonary dysplasia
,
tobacco smoking
,
adrenal cortex neoplasms
,
progression-free survival
,
leukoencephalopathies
,
progressive multifocal leukoencephalopathy
,
histiocytic sarcoma
,
follicular dendritic cell sarcoma
,
interdigitating dendritic cell sarcoma
,
inflammatory breast neoplasms
,
b-cell lymphoma marginal zone
,
bile duct neoplasms
,
carcinoid tumor
,
respiratory tract diseases
,
respiratory tract neoplasms
,
bronchial neoplasms
,
induction chemotherapy
,
nasopharyngeal diseases
,
proton therapy
,
papillomaviridae
,
penile neoplasms
,
papilloma
,
ewing sarcoma
,
esophageal diseases
,
leiomyosarcoma
,
gastrectomy
,
chondrosarcoma
,
adenoviridae infections
,
fever
,
rheumatoid arthritis
,
arthritis
,
alopecia areata
,
alopecia
,
fertility preservation
,
intraoperative complications
,
cachexia
,
maintenance chemotherapy
,
carcinoma
,
neoplasms
,
melanoma
,
colorectal neoplasms
,
liver neoplasms
,
squamous cell carcinoma
,
squamous cell carcinoma of head and neck
,
adenocarcinoma
,
renal cell carcinoma
,
esophageal neoplasms
,
triple negative breast neoplasms
,
head and neck neoplasms
,
esophageal squamous cell carcinoma
,
pancreatic neoplasms
,
small cell lung carcinoma
,
immunotherapy
,
uterine cervical neoplasms
,
ovarian neoplasms
,
neoplasm metastasis
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
urologic neoplasms
,
rectal neoplasms
,
ovarian epithelial carcinoma
,
prostatic neoplasms
,
cholangiocarcinoma
,
squamous cell neoplasms
,
urinary bladder neoplasms
,
large b-cell lymphoma diffuse
,
radiotherapy
,
endometrial neoplasms
,
transitional cell carcinoma
,
skin neoplasms
,
merkel cell carcinoma
,
brain neoplasms
,
anus neoplasms
,
kidney neoplasms
,
drug therapy
,
fallopian tube neoplasms
,
chemoradiotherapy
,
carcinosarcoma
,
mullerian mixed tumor
,
uveal neoplasms
,
hypersensitivity
,
molecular targeted therapy
,
hypopharyngeal neoplasms
,
laryngeal neoplasms
,
transplantation
,
radiation
,
hepatoblastoma
,
hemophagocytic lymphohistiocytosis
,
human herpesvirus 4
,
non-small-cell lung carcinoma
,
lung neoplasms
,
hepatocellular carcinoma
,
stomach neoplasms
,
lymphoma
,
breast neoplasms
,
recurrence
,
hodgkin disease
,
biliary tract neoplasms
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
hepatitis
,
infections
,
hepatitis b
,
chronic hepatitis b
,
chronic hepatitis
,
hepatitis a
,
epstein-barr virus infections
,
virus diseases
,
herpesviridae infections
,
liver diseases
,
neurodegenerative diseases
,
human influenza
,
inflammation
,
alzheimer disease
,
positron-emission tomography
,
tumor microenvironment
,
microbiota
,
metagenome
,
myocarditis
,
interstitial lung diseases
,
disease progression
,
cardiovascular abnormalities
,
anemia
,
hemolytic anemia autoimmune
,
hemolytic anemia
,
vitiligo
,
meningioma
,
uveal melanoma
,
shy-drager syndrome
,
multiple system atrophy
,
atrophy
,
progressive supranuclear palsy
,
movement disorders
,
magnetic resonance imaging
,
x-ray computed tomography
,
dysarthria
,
speech intelligibility
,
immunoblastic lymphadenopathy
,
immune system diseases
,
autoimmune diseases
,
lewy body disease
,
dementia
,
cognitive dysfunction
,
nerve degeneration
,
inborn genetic diseases
,
nervous system diseases
,
nervous system heredodegenerative disorders
,
synucleinopathies
,
irritable bowel syndrome
,
catheter ablation
,
transcriptome
,
retreatment
,
sleep wake disorders
,
dyssomnias
,
circadian rhythm sleep disorders
,
parasomnias
Aspartame
hiv
,
bronchiectasis
,
escherichia coli infections
,
gastroenteritis
,
pharmacokinetics
,
dental plaque
,
hydrogen-ion concentration
,
salivation
,
healthy volunteers/patients
,
xerostomia
,
sjogren's syndrome
,
ototoxicity
,
mucositis
,
glioblastoma
,
pyruvate dehydrogenase complex deficiency disease
,
covid-19
,
sepsis
,
malnutrition
,
child nutrition disorders
,
colonoscopy
,
obesity
,
type 2 diabetes mellitus
,
overweight
,
diabetes mellitus
,
insulin resistance
,
glucose intolerance
,
body weight
,
metabolic syndrome
,
weight loss
,
feeding behavior
,
glucose metabolism disorders
,
metabolic diseases
,
abdominal obesity
,
adiposity
,
dyslipidemias
,
prediabetic state
,
glucose tolerance test
,
cognition
,
fasting
,
type 1 diabetes mellitus
,
placebos
,
hypoglycemia
,
gastric bypass
,
congenital hyperinsulinism
,
nesidioblastosis
,
driving under the influence
,
glycogen storage disease type iii
,
glycogen storage disease
,
kidney transplantation
,
hyperglycemia
,
sleep deprivation
,
cardiovascular diseases
,
mortality
,
hypersensitivity
,
gastrointestinal microbiome
,
chronic disease
,
neoplasms
,
diet
,
adolescent development
,
acromegaly
,
appetitive behavior
,
microbiota
,
chronic periodontitis
,
periodontitis
,
thirst
,
pregnancy rate
,
atherosclerosis
,
appetite
,
intestinal absorption
,
taste
,
hypoxia
,
blood glucose self-monitoring
,
deglutition disorders
,
syndrome
,
thrombosis
,
inflammation
,
platelet aggregation
,
wounds and injuries
,
spinal cord injuries
,
cold temperature
,
sleep
,
blood pressure
,
vascular stiffness
,
aging
,
exercise tolerance
,
alcohol drinking
,
polycystic ovary syndrome
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
,
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
,
healthy volunteers/patients
,
psoriasis
,
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
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
Corticoids
asthma
,
multiple sclerosis
,
relapsing-remitting multiple sclerosis
,
sclerosis
,
smoking
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
PTC518
huntington disease
Mk3475
breast neoplasms
,
prostatic neoplasms
,
renal cell carcinoma
,
melanoma
,
glioma
,
triple negative breast neoplasms
,
hepatocellular carcinoma
,
liver neoplasms
,
uterine cervical neoplasms
,
leiomyosarcoma
,
malignant fibrous histiocytoma
,
sarcoma
,
transitional cell carcinoma
,
urinary bladder neoplasms
,
ureteral neoplasms
,
pancreatic ductal carcinoma
,
colorectal neoplasms
,
carcinoma
,
adenocarcinoma
,
pancreatic neoplasms
,
squamous cell carcinoma of head and neck
,
ovarian neoplasms
,
squamous cell carcinoma
,
biliary tract neoplasms
,
endometrial neoplasms
,
malignant mesothelioma
,
mesothelioma
,
non-hodgkin lymphoma
,
multiple myeloma
,
head and neck neoplasms
,
non-small-cell lung carcinoma
,
neoplasms
,
lung neoplasms
,
recurrence
,
neoplasm metastasis
Solumedrol
uveitis
Docetaxol
renal cell carcinoma
,
pancreatic neoplasms
,
melanoma
,
prostatic neoplasms
,
neoplasms
,
colorectal neoplasms
,
ovarian neoplasms
,
sarcoma
,
synovial sarcoma
,
urethral neoplasms
,
ureteral neoplasms
,
adolescent development
,
castration-resistant prostatic neoplasms
,
stomach neoplasms
,
adenocarcinoma
,
peritoneal neoplasms
,
inflammatory breast neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
carcinoma
,
urologic neoplasms
,
transitional cell carcinoma
,
breast neoplasms
,
peripheral nervous system diseases
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