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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
,
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
,
neuromuscular 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
colorectal neoplasms
,
mesothelioma
,
urinary bladder neoplasms
,
pancreatic ductal carcinoma
,
hepatocellular carcinoma
,
liver neoplasms
,
nasopharyngeal carcinoma
,
uterine cervical neoplasms
,
small cell lung carcinoma
,
esophageal squamous cell carcinoma
,
nasopharyngeal neoplasms
,
malignant mesothelioma
,
stomach neoplasms
,
castration-resistant prostatic neoplasms
,
ovarian neoplasms
,
triple negative breast neoplasms
,
basal cell carcinoma
,
urologic neoplasms
,
hereditary sensory and autonomic neuropathies
,
neoplasms
,
pancreatic neoplasms
,
merkel cell carcinoma
,
melanoma
,
glioblastoma
,
endometrial neoplasms
,
adenocarcinoma
,
liposarcoma
,
hodgkin disease
,
glioma
,
astrocytoma
,
oligodendroglioma
,
sarcoma
,
microsatellite instability
,
exanthema
,
non-small-cell lung carcinoma
,
carcinoma
,
squamous cell carcinoma
,
squamous cell carcinoma of head and neck
,
lung neoplasms
,
renal cell carcinoma
,
head and neck neoplasms
,
neoplasm metastasis
,
transitional cell carcinoma
,
anus neoplasms
Alfacalcidol
asthma
,
healthy volunteers/patients
,
non-small-cell lung carcinoma
,
lung injury
,
lung neoplasms
,
smoking
,
family relations
Aspartame
medulloblastoma
,
ependymoma
Bevifimod
alzheimer disease
,
macular degeneration
,
hepatic insufficiency
,
cognitive dysfunction
,
immunologic deficiency syndromes
,
precursor cell lymphoblastic leukemia-lymphoma
,
maximum tolerated dose
,
antineoplastic protocols
,
pharmacokinetics
,
myeloproliferative disorders
,
type 1 diabetes mellitus
,
ichthyosis
,
acquired immunodeficiency syndrome
,
hiv
,
aging
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
adenocarcinoma of lung
,
hematologic neoplasms
,
biliary liver cirrhosis
,
neonatal anemia
,
peripheral nervous system diseases
,
arthroscopy
,
critical illness
,
gastrointestinal diseases
,
hidradenitis suppurativa
,
hepatitis b
,
chronic hepatitis b
,
hepatitis
,
hepatitis a
,
syndrome
,
glioblastoma
,
glioma
,
gliosarcoma
,
astrocytoma
,
diffuse intrinsic pontine glioma
,
optic nerve glioma
,
brain stem neoplasms
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
pathologic complete response
,
retroviridae infections
,
viral sexually transmitted diseases
,
pancreatic neoplasms
,
triple negative breast neoplasms
,
sickle cell anemia
,
immunoglobulin light-chain amyloidosis
,
healthy volunteers/patients
,
multiple myeloma
,
prostatic neoplasms
,
non-small-cell lung carcinoma
,
large b-cell lymphoma diffuse
,
orthopedic procedures
,
plasma cell neoplasms
,
rheumatoid arthritis
,
b-cell lymphoma
,
chronic renal insufficiency
,
blood transfusion
,
non-hodgkin lymphoma
,
psoriatic arthritis
,
recurrence
,
mantle-cell lymphoma
,
kidney diseases
,
chronic kidney failure
,
systemic lupus erythematosus
,
follicular lymphoma
,
b-cell chronic lymphocytic leukemia
,
myelodysplastic syndromes
,
breast neoplasms
,
autologous blood transfusion
,
diabetes mellitus
,
hemorrhage
,
colorectal neoplasms
,
ankylosing spondylitis
,
ovarian neoplasms
,
axial spondyloarthritis
,
leukemia
,
lymphoid leukemia
,
renal insufficiency
,
drug therapy
,
neoplasm metastasis
,
cardiovascular surgical procedures
,
cerebrospinal fluid leak
,
peripheral vascular diseases
,
respiratory syncytial viruses
,
juvenile arthritis
,
waldenstrom macroglobulinemia
,
b-cell lymphoma marginal zone
,
skin diseases
,
colitis
,
retinal diseases
,
esophageal neoplasms
,
small cell lung carcinoma
,
mammaplasty
,
venous thrombosis
,
stomach neoplasms
,
rectal neoplasms
,
diabetic foot
,
diabetic neuropathies
,
foot ulcer
,
skin ulcer
,
ebola hemorrhagic fever
,
soft tissue injuries
,
surgical blood loss
,
neurologic manifestations
,
respiratory tract diseases
,
covid-19
,
kidney transplantation
,
neonatal alloimmune thrombocytopenia
,
pouchitis
,
anemia
,
neoplasms
,
psoriasis
,
crohn disease
,
lymphoma
,
ulcerative colitis
,
type 2 diabetes mellitus
,
inflammatory bowel diseases
,
hiv infections
,
cardiovascular diseases
,
obesity
,
atrial fibrillation
,
metabolic syndrome
,
pure red-cell aplasia
,
postoperative pain
,
abdominal hernia
,
hernia
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
atrophic vaginitis
,
physiological sexual dysfunction
,
psychological sexual dysfunctions
,
breast neoplasms
,
hot flashes
,
dyspareunia
,
menopause
Lactams
acquired immunodeficiency syndrome
,
drug interactions
,
hiv infections
,
healthy volunteers/patients
,
vomiting
,
nausea
,
tuberculosis
,
pulmonary tuberculosis
,
intraabdominal infections
,
abscess
,
peritonitis
,
bacillary dysentery
,
ulcer
,
buruli ulcer
,
neoplasms
,
pyelonephritis
,
gonorrhea
,
sinusitis
,
rhinosinusitis
,
neutropenia
,
fever
,
infections
,
respiratory tract infections
,
bacterial infections
,
communicable diseases
,
urinary tract infections
,
otitis media
,
otitis
,
ventilator-associated pneumonia
,
pneumonia
,
dental focal infection
,
focal infection
,
cystic fibrosis
,
pseudomonas infections
,
pseudomonas aeruginosa
,
postoperative complications
,
surgical wound infection
,
infectious skin diseases
,
skin diseases
,
soft tissue infections
,
sickle cell anemia
,
yin deficiency
Mk3475
pancreatic neoplasms
,
pancreatic ductal carcinoma
,
non-small-cell lung carcinoma
,
neoplasms
,
mesothelioma
,
colorectal neoplasms
,
gastrointestinal neoplasms
,
lung neoplasms
,
neoplasm metastasis
Solumedrol
graft rejection
,
organ transplantation
,
psychology rejection
,
melanoma
,
leukemia
,
myelodysplastic syndromes
,
myeloid leukemia acute
,
myeloid leukemia
,
preleukemia
,
syndrome
,
renal cell carcinoma
,
lung neoplasms
,
synovitis
,
lymphoma
,
t-cell lymphoma
,
t-cell lymphoma peripheral
,
immunoglobulin g4-related disease
,
systemic lupus erythematosus
,
polymyalgia rheumatica
,
giant cell arteritis
,
kidney transplantation
,
epstein-barr virus infections
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