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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
,
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
tic disorders
,
tourette syndrome
,
tics
,
alzheimer disease
,
dementia
,
muscular diseases
,
rett syndrome
,
ataxia
,
inborn genetic diseases
,
retinal diseases
,
homocystinuria
,
syndrome
,
inborn errors lipid metabolism
,
noise-induced hearing loss
,
angelman syndrome
,
autistic disorder
,
autism spectrum disorder
,
cerebellar ataxia
,
parkinson disease
,
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
,
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
squamous cell carcinoma of head and neck
,
neoplasms
,
non-small-cell lung carcinoma
,
neoplasm metastasis
,
carcinoma
,
anus neoplasms
,
squamous cell carcinoma
,
colorectal neoplasms
,
triple negative breast neoplasms
,
sarcoma
,
biliary tract neoplasms
,
tertiary lymphoid structures
Alfacalcidol
carcinoma
,
squamous cell carcinoma
,
squamous cell neoplasms
,
atopic dermatitis
,
eczema
,
dermatitis
Aspartame
healthy volunteers/patients
,
renal insufficiency
,
chronic kidney failure
,
myeloid leukemia acute
,
immunoassay
,
myelodysplastic syndromes
,
non-hodgkin lymphoma
,
hiv
,
acquired immunodeficiency syndrome
,
immunologic deficiency syndromes
,
acute disease
,
virus diseases
,
respiratory syncytial virus infections
,
leukemia
,
precursor cell lymphoblastic leukemia-lymphoma
,
respiratory syncytial viruses
,
venous thrombosis
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
lymphoid leukemia
,
smoldering multiple myeloma
,
hiv-1
,
neoplasm metastasis
,
chronic hepatitis c
,
breast neoplasms
,
postmenopause
,
peripheral nervous system diseases
,
hematologic neoplasms
,
colorectal neoplasms
,
myositis
,
graft vs host disease
,
bronchiolitis obliterans syndrome
,
multiple myeloma
,
psoriasis
,
non-small-cell lung carcinoma
,
hiv infections
,
plasma cell neoplasms
,
neoplasms
,
infections
,
communicable diseases
,
heart failure
,
covid-19
,
stroke
,
lymphoma
,
ovarian neoplasms
,
juvenile arthritis
,
thromboembolism
,
respiratory tract infections
,
depression
,
major depressive disorder
,
depressive disorder
,
treatment-resistant depressive disorder
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
ovarian epithelial carcinoma
,
inflammatory bowel diseases
,
b-cell chronic lymphocytic leukemia
,
recurrence
,
respiratory insufficiency
,
rheumatic diseases
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
tinea pedis
,
psychotic disorders
,
bipolar disorder
,
psychotic affective disorders
,
mood disorders
,
mental disorders
,
human influenza
,
venous thromboembolism
,
kidney transplantation
,
schizophrenia
,
type 2 diabetes mellitus
,
crohn disease
,
prostatic neoplasms
,
urogenital neoplasms
,
male genital diseases
,
male genital neoplasms
,
genital diseases
,
lung neoplasms
,
hyperkinesis
,
attention deficit disorder with hyperactivity
,
cognitive dysfunction
,
thyroid neoplasms
,
papillary thyroid cancer
,
thyroid diseases
,
goiter
,
obesity
,
retinal diseases
,
ankle injuries
,
ankle fractures
,
fibula fractures
Bevifimod
small cell lung carcinoma
,
glioblastoma
,
merkel cell carcinoma
,
non-muscle invasive bladder neoplasms
,
burkitt lymphoma
,
pancreatic ductal carcinoma
,
neurofibromatosis 1
,
hereditary sensory and autonomic neuropathies
,
central nervous system neoplasms
,
brain diseases
,
neuroblastoma
,
ewing sarcoma
,
drug interactions
,
transitional cell carcinoma
,
uterine cervical neoplasms
,
urinary bladder neoplasms
,
thyroid neoplasms
,
brain concussion
,
post-concussion syndrome
,
astrocytoma
,
myeloproliferative disorders
,
b-cell chronic lymphocytic leukemia
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
t-cell lymphoma cutaneous
,
sarcoma
,
mycosis fungoides
,
squamous cell neoplasms
,
postoperative pain
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
lyme neuroborreliosis
,
ocular hypertension
,
open-angle glaucoma
,
triple negative breast neoplasms
,
melanoma
,
multiple myeloma
,
carcinoma
,
brain neoplasms
,
recurrence
,
respiratory syncytial viruses
,
hepatocellular carcinoma
,
plasma cell neoplasms
,
head and neck neoplasms
,
ovarian neoplasms
,
glioma
,
hodgkin disease
,
endometrial neoplasms
,
liver neoplasms
,
squamous cell carcinoma
,
atopic dermatitis
,
paraproteinemias
,
pancreatic neoplasms
,
osteoporosis
,
ovarian epithelial carcinoma
,
esophageal neoplasms
,
hematopoietic stem cell transplantation
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
human influenza
,
oligodendroglioma
,
myelodysplastic syndromes
,
inflammation
,
macular edema
,
diabetic retinopathy
,
eczema
,
skin diseases
,
plasmacytoma
,
plasma cell leukemia
,
disease progression
,
motor activity
,
overactive urinary bladder
,
keratoconjunctivitis sicca
,
dry eye syndromes
,
communicable diseases
,
interstitial cystitis
,
meningococcal infections
,
meningococcal meningitis
,
meningitis
,
serogroup b neisseria meningitidis
,
hematologic diseases
,
immune system diseases
,
blood protein disorders
,
lymphoproliferative disorders
,
vascular diseases
,
neoplasms by histologic type
,
immunoproliferative disorders
,
hemorrhagic disorders
,
asthma
,
waldenstrom macroglobulinemia
,
skin neoplasms
,
myeloid leukemia
,
malignant mesothelioma
,
mesothelioma
,
sickle cell anemia
,
hemolysis
,
monocytic leukemia acute
,
irritable bowel syndrome
,
facial pain
,
small fiber neuropathy
,
myelomonocytic leukemia chronic
,
discoid lupus erythematosus
,
cutaneous lupus erythematosus
,
meningeal neoplasms
,
breast neoplasms
,
colorectal neoplasms
,
non-small-cell lung carcinoma
,
lymphoma
,
myeloid leukemia acute
,
renal cell carcinoma
,
prostatic neoplasms
,
colonic neoplasms
,
squamous cell carcinoma of head and neck
,
neoplasm metastasis
,
coronavirus infections
,
non-hodgkin lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
rectal neoplasms
,
psoriatic arthritis
,
follicular lymphoma
,
infections
,
lyme disease
,
adenocarcinoma
,
juvenile arthritis
,
lung neoplasms
,
kidney neoplasms
,
borrelia infections
,
rectal diseases
,
crohn disease
,
neuralgia
,
partial epilepsies
,
male breast neoplasms
,
respiratory tract infections
,
stomach neoplasms
,
bacterial infections
,
infectious skin diseases
,
wound infection
,
soft tissue infections
,
cellulitis
,
gastrointestinal neoplasms
,
digestive system neoplasms
,
gastrointestinal diseases
,
intestinal neoplasms
,
digestive system diseases
,
colonic diseases
,
intestinal diseases
,
pruritus
,
chronic renal insufficiency
,
chronic kidney failure
,
obesity
,
endocrine system diseases
,
fetal growth retardation
,
growth disorders
,
hematologic neoplasms
,
invasive pulmonary aspergillosis
,
neuroaspergillosis
,
neutropenia
,
fever
,
febrile neutropenia
,
hyperthermia
,
amyloid plaque
,
bronchiolitis obliterans syndrome
,
lymphoid leukemia
,
restless legs syndrome
,
sleep deprivation
,
complex partial epilepsy
,
metabolic syndrome
,
abdominal obesity
,
pneumococcal infections
,
vector borne diseases
,
immunization
,
residual neoplasm
,
carpal tunnel syndrome
,
aplastic anemia
,
polyneuropathies
,
delirium
,
liver cirrhosis
,
muscle cramp
,
lung diseases
,
virus diseases
,
coronaviridae infections
,
neoplasms by site
,
rheumatoid arthritis
,
neoplasms
,
healthy volunteers/patients
,
covid-19
,
hemophilia a
,
leukemia
,
psoriasis
,
candidemia
,
precursor cell lymphoblastic leukemia-lymphoma
,
type 2 diabetes mellitus
,
hemophilia b
,
pain
,
ankylosing spondylitis
,
pituitary dwarfism
,
candidiasis
,
aspergillosis
,
invasive candidiasis
,
diabetes mellitus
,
arthritis
,
kidney transplantation
,
precursor b-cell lymphoblastic leukemia-lymphoma
,
schizophrenia
,
laron syndrome
,
respiratory tract diseases
,
mycoses
,
pharmacokinetics
,
pneumonia
,
postherpetic neuralgia
,
cardiovascular diseases
,
axial spondyloarthritis
,
brain injuries
,
type 1 diabetes mellitus
,
insulin resistance
,
hemostatic disorders
,
diabetic neuropathies
,
anxiety disorders
,
ventilator-associated pneumonia
,
relapsing-remitting multiple sclerosis
,
tremor
,
essential tremor
,
atherosclerosis
,
non-radiographic axial spondyloarthritis
,
electrocardiography
,
smoking cessation
,
traumatic brain injuries
,
dwarfism
,
self concept
,
pathologic constriction
,
spinal stenosis
,
abdominal pain
,
tissue adhesions
,
fibromyalgia
,
sepsis
,
obsessive-compulsive disorder
,
rheumatic fever
,
liver diseases
,
agnosia
,
anxiety
,
neisseria meningitidis
,
ulcerative colitis
,
inflammatory bowel diseases
,
fatty liver
,
metabolism
,
metabolic bone diseases
,
healthcare-associated pneumonia
,
drug-related side effects and adverse reactions
,
sars-cov-2
,
uremia
,
hypercholesterolemia
,
coronary disease
,
coronary artery disease
,
myocardial ischemia
,
heart failure
,
amyloidosis
,
vaccination
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
,
covid-19
,
takayasu arteritis
,
giant cell arteritis
,
crohn disease
,
eczema
,
dermatitis
,
ulcerative colitis
,
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
renal cell carcinoma
,
neoplasms
,
non-small-cell lung carcinoma
,
urinary bladder neoplasms
,
melanoma
,
oncogenes
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
1
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