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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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Marketed Drugs
Therapeutic Areas:
All
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
,
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
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
lymphoma
,
hodgkin disease
,
non-small-cell lung carcinoma
,
liver neoplasms
,
lung neoplasms
,
hepatocellular carcinoma
,
stomach neoplasms
,
adenocarcinoma
,
colorectal neoplasms
,
carcinoma
,
squamous cell carcinoma of head and neck
,
squamous cell carcinoma
,
neoplasm metastasis
,
melanoma
,
sarcoma
,
urologic neoplasms
,
transitional cell carcinoma
,
biliary tract neoplasms
,
second primary neoplasms
,
colonic neoplasms
,
rectal neoplasms
,
esophageal neoplasms
,
head and neck neoplasms
,
cholangiocarcinoma
,
pancreatic ductal carcinoma
,
renal cell carcinoma
,
pancreatic neoplasms
,
leiomyosarcoma
,
ovarian neoplasms
,
endometrial neoplasms
,
tertiary lymphoid structures
,
brain neoplasms
,
breast neoplasms
,
neoplasms
,
triple negative breast neoplasms
Aspartame
hyperlipidemias
,
gastrointestinal stromal tumors
,
anemia
,
iron-deficiency anemia
,
iron deficiencies
,
glioblastoma
,
gliosarcoma
,
lung neoplasms
,
small cell lung carcinoma
,
healthy volunteers/patients
,
leukemia
,
primary myelofibrosis
,
myeloid leukemia acute
,
myeloid leukemia
,
carcinoma
,
hepatocellular carcinoma
,
diarrhea
,
plexiform neurofibroma
,
neurofibromatoses
,
neurofibroma
,
thalassemia
,
iron overload
,
chronic obstructive pulmonary disease
,
obesity
,
triple negative breast neoplasms
,
pre-eclampsia
,
eclampsia
,
essential thrombocythemia
,
polycythemia vera
,
thrombocytosis
,
polycythemia
,
lupus nephritis
,
nephritis
,
inflammatory breast neoplasms
,
prostatic neoplasms
,
precursor cell lymphoblastic leukemia-lymphoma
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
b-cell chronic lymphocytic leukemia
,
lymphoid leukemia
,
hemorrhage
,
wiskott-aldrich syndrome
,
bcr-abl positive chronic myelogenous leukemia
,
metaplasia
,
graft vs host disease
,
hematopoietic stem cell transplantation
,
bronchiolitis obliterans syndrome
,
syndrome
,
hyperlipoproteinemia type ii
,
organ transplantation
,
homozygous familial hypercholesterolemia
,
cryopyrin-associated periodic syndromes
,
myelodysplastic syndromes
,
preleukemia
,
liver transplantation
,
thrombocytopenic purpura idiopathic
,
thrombocytopenic purpura
,
purpura
,
parkinson disease
,
eosinophilia
,
cellulitis
,
juvenile arthritis
,
arthritis
,
hepatitis b
,
hepatitis
,
hepatitis b virus
,
hepatitis a
,
macular edema
,
edema
,
vision disorders
,
low vision
,
pituitary acth hypersecretion
,
acth-secreting pituitary adenoma
,
osteoporosis
,
acute coronary syndrome
,
bronchitis
,
acute disease
,
pulmonary eosinophilia
,
pancreatic neoplasms
,
fistula
,
delirium
,
kidney transplantation
,
asthma
,
hypercholesterolemia
,
neoplasms
,
cardiovascular diseases
,
atherosclerosis
,
chronic kidney failure
,
kidney diseases
,
thrombocytopenia
,
breast neoplasms
,
heart failure
,
renal insufficiency
,
infections
,
dyslipidemias
,
chagas disease
,
chronic renal insufficiency
,
renal dialysis
,
renal replacement therapy
,
drug-related side effects and adverse reactions
,
heart transplantation
,
polycystic kidney diseases
,
autosomal dominant polycystic kidney
,
arthrogryposis
,
fibrosis
,
cystic fibrosis
,
pseudomonas infections
,
type 2 diabetes mellitus
,
diabetes mellitus
,
multiple myeloma
,
plasma cell neoplasms
,
transplantation
,
cytomegalovirus infections
,
communicable diseases
,
metabolic syndrome
,
preconception care
,
drug interactions
,
melanoma
,
schizophrenia
Bevifimod
multiple sclerosis
,
sclerosis
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
neoplasms
PTC518
huntington disease
Mk3475
thoracic neoplasms
,
glandular and epithelial neoplasms
,
lip neoplasms
,
eye neoplasms
,
myelodysplastic syndromes
,
pharmacokinetics
,
soft tissue neoplasms
,
digestive system neoplasms
,
lung diseases
,
respiratory tract neoplasms
,
bronchogenic carcinoma
,
endocrine gland neoplasms
,
breast diseases
,
bronchial neoplasms
,
intraductal carcinoma noninfiltrating
,
myoepithelioma
,
oligodendroglioma
,
death
,
pancreatic neoplasms
,
neoplasm metastasis
,
sarcoma
,
b-cell lymphoma
,
castration-resistant prostatic neoplasms
,
brain neoplasms
,
large b-cell lymphoma diffuse
,
non-hodgkin lymphoma
,
t-cell lymphoma
,
leukemia
,
neuroendocrine tumors
,
cholangiocarcinoma
,
follicular lymphoma
,
myeloid leukemia acute
,
neuroendocrine carcinoma
,
pancreatic ductal carcinoma
,
t-cell lymphoma peripheral
,
kidney neoplasms
,
thyroid neoplasms
,
uterine neoplasms
,
uveal neoplasms
,
myeloid leukemia
,
invasive hydatidiform mole
,
lymphoid leukemia
,
glioma
,
female genital neoplasms
,
small cell carcinoma
,
urethral neoplasms
,
ureteral neoplasms
,
colonic neoplasms
,
vulvar neoplasms
,
b-cell lymphoma marginal zone
,
anus neoplasms
,
thyroid diseases
,
cutaneous malignant melanoma
,
thymoma
,
microsatellite repeats
,
gastrointestinal neoplasms
,
mouth neoplasms
,
oropharyngeal neoplasms
,
laryngeal neoplasms
,
salivary gland neoplasms
,
endometrioid carcinoma
,
serous cystadenocarcinoma
,
mantle-cell lymphoma
,
waldenstrom macroglobulinemia
,
penile neoplasms
,
b-cell chronic lymphocytic leukemia
,
gliosarcoma
,
carcinosarcoma
,
mullerian mixed tumor
,
astrocytoma
,
thymus neoplasms
,
rectal neoplasms
,
hypopharyngeal neoplasms
,
male genital neoplasms
,
bile duct neoplasms
,
respiratory tract infections
,
papillomavirus infections
,
papilloma
,
adenoid cystic carcinoma
,
meningioma
,
male breast neoplasms
,
leiomyosarcoma
,
malignant fibrous histiocytoma
,
epstein-barr virus infections
,
adenocarcinoma of lung
,
oncogenes
,
neurofibrosarcoma
,
nerve sheath neoplasms
,
mycoses
,
sezary syndrome
,
mycosis fungoides
,
t-cell lymphoma cutaneous
,
syndrome
,
pelvic neoplasms
,
hemangiosarcoma
,
osteosarcoma
,
germ cell and embryonal neoplasms
,
neoplasms by histologic type
,
adenoviridae infections
,
basal cell carcinoma
,
anaplastic thyroid carcinoma
,
reactive arthritis
,
gallbladder neoplasms
,
clear cell adenocarcinoma
,
adenosquamous carcinoma
,
gestational trophoblastic disease
,
alveolar soft part sarcoma
,
precursor cell lymphoblastic leukemia-lymphoma
,
biphenotypic leukemia acute
,
local neoplasm recurrence
,
sclerosis
,
chordoma
,
meningeal carcinomatosis
,
papillomaviridae
,
second primary neoplasms
,
immunoblastic lymphadenopathy
,
enteropathy-associated t-cell lymphoma
,
intestinal diseases
,
panniculitis
,
carcinoma in situ
,
cervical intraepithelial neoplasia
,
uterine cervical dysplasia
,
squamous intraepithelial lesions of the cervix
,
central nervous system diseases
,
extranodal nk-t-cell lymphoma
,
liposarcoma
,
virus diseases
,
viremia
,
unknown primary neoplasms
,
synovial sarcoma
,
point mutation
,
oral leukoplakia
,
leukoplakia
,
lymphoproliferative disorders
,
endoscopy
,
colonic diseases
,
histiocytic sarcoma
,
follicular dendritic cell sarcoma
,
interdigitating dendritic cell sarcoma
,
environmental biomarkers
,
circulating neoplastic cells
,
inflammatory breast neoplasms
,
meningeal neoplasms
,
intestinal neoplasms
,
central nervous system neoplasms
,
nervous system neoplasms
,
nervous system diseases
,
nerve tissue neoplasms
,
primitive neuroectodermal tumors
,
neuroepithelial neoplasms
,
neuroectodermal tumors
,
adrenocortical carcinoma
,
carcinoid tumor
,
mediastinal neoplasms
,
testicular neoplasms
,
gastrointestinal stromal tumors
,
hairy cell leukemia
,
burkitt lymphoma
,
laryngeal diseases
,
vaginal neoplasms
,
acinar cell carcinoma
,
adenomyoepithelioma
,
hereditary breast and ovarian cancer syndrome
,
maxillary sinus neoplasms
,
basal cell neoplasms
,
urogenital neoplasms
,
male urogenital diseases
,
urogenital diseases
,
neoplasms
,
lung neoplasms
,
breast neoplasms
,
colorectal neoplasms
,
triple negative breast neoplasms
,
lymphoma
,
renal cell carcinoma
,
ovarian neoplasms
,
adenocarcinoma
,
recurrence
,
urinary bladder neoplasms
,
head and neck neoplasms
,
prostatic neoplasms
,
esophageal neoplasms
,
stomach neoplasms
,
liver neoplasms
,
hepatocellular carcinoma
,
endometrial neoplasms
,
uterine cervical neoplasms
,
ovarian epithelial carcinoma
,
small cell lung carcinoma
,
transitional cell carcinoma
,
hodgkin disease
,
fallopian tube neoplasms
,
urologic neoplasms
,
multiple myeloma
,
glioblastoma
,
squamous cell neoplasms
,
mesothelioma
,
plasma cell neoplasms
,
skin neoplasms
,
esophageal squamous cell carcinoma
,
biliary tract neoplasms
,
malignant mesothelioma
,
peritoneal neoplasms
,
microsatellite instability
,
merkel cell carcinoma
,
non-muscle invasive bladder neoplasms
,
hematologic neoplasms
,
hypersensitivity
,
nasopharyngeal neoplasms
,
nasopharyngeal carcinoma
,
disease progression
,
muscle neoplasms
,
carcinoma
,
non-small-cell lung carcinoma
,
melanoma
,
squamous cell carcinoma of head and neck
,
squamous cell carcinoma
Solumedrol
macular degeneration
,
postmenopause
,
prostatic neoplasms
,
castration-resistant prostatic neoplasms
,
multiple myeloma
,
plasma cell neoplasms
,
breast neoplasms
,
neoplasms
,
urogenital neoplasms
,
male genital diseases
,
male genital neoplasms
,
genital diseases
Docetaxol
paraneoplastic syndromes
,
ovarian neoplasms
,
esophageal neoplasms
,
pancreatic neoplasms
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
ovarian epithelial carcinoma
,
inflammatory breast neoplasms
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
urinary bladder neoplasms
,
diarrhea
,
hepatocellular carcinoma
,
liver neoplasms
,
gastrointestinal diseases
,
digestive system diseases
,
ductal carcinoma
,
mouth neoplasms
,
endometrioid carcinoma
,
endometrial neoplasms
,
hypersensitivity
,
uterine neoplasms
,
breast neoplasms
,
stomach neoplasms
,
squamous cell carcinoma
,
carcinoma
,
adenocarcinoma
,
squamous cell carcinoma of head and neck
,
squamous cell neoplasms
,
recurrence
,
neoplasm metastasis
,
castration-resistant prostatic neoplasms
,
laryngeal neoplasms
,
laryngeal diseases
,
brain stem neoplasms
,
local neoplasm recurrence
,
hypopharyngeal neoplasms
,
prostatic neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
neoplasms
,
head and neck neoplasms
,
neutropenia
,
alopecia areata
,
alopecia
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