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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
,
limb-girdle muscular dystrophies
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
castration-resistant prostatic neoplasms
,
virus diseases
,
herpesviridae infections
,
prostatic neoplasms
,
hepatitis b
,
chronic hepatitis b
,
hepatitis
,
hepatitis a
,
neoplasms
,
leukemia
,
hematologic diseases
,
b-cell chronic lymphocytic leukemia
,
bone marrow diseases
,
carcinoma
,
small cell carcinoma
,
small cell lung carcinoma
,
neuroendocrine carcinoma
,
colorectal neoplasms
,
colonic neoplasms
,
colonic diseases
,
multiple myeloma
,
plasma cell neoplasms
Alfacalcidol
asthma
,
non-small-cell lung carcinoma
,
lung injury
,
lung neoplasms
,
smoking
,
family relations
Aspartame
lymphoma
,
conjunctivitis
,
allergic rhinitis
,
rhinitis
,
allergic conjunctivitis
,
liver neoplasms
,
atopic dermatitis
,
dermatitis
,
neoplasm metastasis
,
healthy volunteers/patients
,
neoplasms
,
prostatic neoplasms
,
vomiting
,
nausea
,
infections
,
esophageal neoplasms
,
postoperative nausea and vomiting
,
brain neoplasms
,
second primary neoplasms
,
depression
,
treatment-resistant depressive disorder
,
depressive disorder
,
esophageal squamous cell carcinoma
,
hematologic neoplasms
,
pneumonia
,
pseudomonas infections
,
pseudomonas aeruginosa
,
bacteremia
,
renal cell carcinoma
,
liver cirrhosis
,
experimental liver cirrhosis
,
chronic obstructive pulmonary disease
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
recurrence
,
melanoma
,
colorectal neoplasms
,
pancreatic ductal carcinoma
,
biliary tract neoplasms
,
non-muscle invasive bladder neoplasms
,
non-small-cell lung carcinoma
,
hiv infections
,
carcinoma
,
acquired immunodeficiency syndrome
,
lung neoplasms
,
ovarian neoplasms
,
uterine cervical neoplasms
,
triple negative breast neoplasms
,
breast neoplasms
,
condylomata acuminata
,
fibrosis
,
hepatocellular carcinoma
,
hiv-2
,
immunologic deficiency syndromes
,
hiv-1
,
antibiotic prophylaxis
,
hepatitis b
,
chronic hepatitis b
,
ovarian epithelial carcinoma
,
coronary disease
,
coronary artery disease
,
myocardial ischemia
,
heart diseases
,
lung diseases
,
cystic fibrosis
,
pulmonary hypertension
,
pulmonary arterial hypertension
,
endometrial neoplasms
,
fallopian tube neoplasms
,
asthma
,
chronic hepatitis c
,
hepatitis
,
hepatitis a
,
hepatitis c
,
hiv
,
neuromuscular blockade
,
chronic kidney failure
,
kidney diseases
,
chronic hepatitis
,
intestinal diseases
,
hypertension
,
non-hodgkin lymphoma
,
infertility
,
schizophrenia
,
traumatic brain injuries
,
brain concussion
,
brain injuries
,
post-concussion syndrome
,
papillomavirus infections
,
uterine cervical dysplasia
Bevifimod
psoriasis
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
healthy volunteers/patients
,
breast neoplasms
,
renal cell carcinoma
,
brain neoplasms
,
ovarian neoplasms
,
pancreatic neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
kidney neoplasms
,
atopic dermatitis
,
eczema
,
dermatitis
,
immune system diseases
,
inflammation
,
bronchial diseases
,
hypersensitivity
,
immediate hypersensitivity
,
respiratory hypersensitivity
,
colorectal neoplasms
,
gastroesophageal reflux
,
bullous pemphigoid
,
churg-strauss syndrome
,
nasal polyps
,
nasopharyngeal neoplasms
,
eosinophilic esophagitis
,
peptic esophagitis
,
esophagitis
,
asthma
,
chronic obstructive pulmonary disease
,
obstructive lung diseases
,
lung diseases
,
systemic lupus erythematosus
,
pulmonary eosinophilia
,
respiratory tract diseases
,
eosinophilia
,
respiration disorders
,
emphysema
,
respiratory sounds
,
disease progression
,
allergic rhinitis
,
rhinitis
,
allergic rhinitis perennial
,
hypercholesterolemia
,
metabolic bone diseases
,
osteoporosis
,
bone fractures
,
osteoporotic fractures
Lactams
renal insufficiency
,
breast neoplasms
,
overactive urinary bladder
,
soft tissue infections
,
pelvic infection
,
urologic diseases
,
hematologic neoplasms
,
neoplasms
,
urinary tract infections
,
pyelonephritis
,
surgical wound infection
,
bacterial pneumonia
,
bacterial infections
,
diabetic foot
,
bacteremia
,
infections
,
communicable diseases
,
intraabdominal infections
,
sepsis
,
pneumonia
,
healthy volunteers/patients
,
febrile neutropenia
,
tuberculosis
,
endometritis
,
puerperal infection
,
prostatic hyperplasia
,
hypersensitivity
,
immediate hypersensitivity
,
chronic kidney failure
,
continuous ambulatory peritoneal dialysis
,
neutropenia
,
fever
,
hyperthermia
,
ventilator-associated pneumonia
,
contraception
,
critical illness
,
obesity
Mk3475
lung diseases
,
myelodysplastic syndromes
,
lymphoid leukemia
,
thoracic neoplasms
,
neurologic manifestations
,
papillomaviridae
,
pharmacokinetics
,
soft tissue neoplasms
,
precursor cell lymphoblastic leukemia-lymphoma
,
respiratory tract neoplasms
,
bronchogenic carcinoma
,
endocrine gland neoplasms
,
breast diseases
,
bronchial neoplasms
,
intraductal carcinoma noninfiltrating
,
laryngeal diseases
,
myoepithelioma
,
oligodendroglioma
,
lymphoma
,
pancreatic neoplasms
,
sarcoma
,
brain neoplasms
,
castration-resistant prostatic neoplasms
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
non-hodgkin lymphoma
,
t-cell lymphoma
,
myeloid leukemia acute
,
follicular lymphoma
,
leukemia
,
neuroendocrine tumors
,
pancreatic ductal carcinoma
,
t-cell lymphoma peripheral
,
kidney neoplasms
,
microsatellite instability
,
uveal neoplasms
,
glioma
,
invasive hydatidiform mole
,
neuroendocrine carcinoma
,
thyroid neoplasms
,
myeloid leukemia
,
urethral neoplasms
,
colonic neoplasms
,
uterine neoplasms
,
uveal melanoma
,
gastrointestinal neoplasms
,
mouth neoplasms
,
oropharyngeal neoplasms
,
small cell carcinoma
,
vulvar neoplasms
,
b-cell lymphoma marginal zone
,
anus neoplasms
,
female genital neoplasms
,
hypersensitivity
,
thymoma
,
epstein-barr virus infections
,
microsatellite repeats
,
rectal neoplasms
,
hypopharyngeal neoplasms
,
salivary gland neoplasms
,
endometrioid carcinoma
,
serous cystadenocarcinoma
,
mantle-cell lymphoma
,
waldenstrom macroglobulinemia
,
penile neoplasms
,
neoplasms by histologic type
,
glandular and epithelial neoplasms
,
liposarcoma
,
b-cell chronic lymphocytic leukemia
,
t-cell lymphoma cutaneous
,
ureteral neoplasms
,
gliosarcoma
,
thyroid diseases
,
carcinosarcoma
,
astrocytoma
,
thymus neoplasms
,
burkitt lymphoma
,
laryngeal neoplasms
,
disease
,
adenoid cystic carcinoma
,
meningioma
,
male breast neoplasms
,
adenocarcinoma of lung
,
oncogenes
,
neurofibrosarcoma
,
nerve sheath neoplasms
,
mycoses
,
sezary syndrome
,
mycosis fungoides
,
syndrome
,
pelvic neoplasms
,
osteosarcoma
,
central nervous system neoplasms
,
germ cell and embryonal neoplasms
,
digestive system neoplasms
,
basal cell carcinoma
,
anaplastic thyroid carcinoma
,
reactive arthritis
,
mullerian mixed tumor
,
adenosquamous carcinoma
,
gestational trophoblastic disease
,
alveolar soft part sarcoma
,
biphenotypic leukemia acute
,
local neoplasm recurrence
,
sclerosis
,
chordoma
,
meningeal carcinomatosis
,
immunoblastic lymphadenopathy
,
enteropathy-associated t-cell lymphoma
,
vaccinia
,
carcinoma in situ
,
cervical intraepithelial neoplasia
,
uterine cervical dysplasia
,
squamous intraepithelial lesions of the cervix
,
disease progression
,
central nervous system diseases
,
extranodal nk-t-cell lymphoma
,
neoplasms by site
,
x-linked bulbo-spinal atrophy
,
leiomyosarcoma
,
malignant fibrous histiocytoma
,
nasopharyngeal carcinoma
,
virus diseases
,
viremia
,
unknown primary neoplasms
,
synovial sarcoma
,
point mutation
,
oral leukoplakia
,
leukoplakia
,
hematologic diseases
,
bone marrow diseases
,
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
,
nervous system neoplasms
,
kidney diseases
,
nervous system diseases
,
nerve tissue neoplasms
,
neuroepithelial neoplasms
,
neuroectodermal tumors
,
adrenocortical carcinoma
,
carcinoid tumor
,
parotid neoplasms
,
mediastinal neoplasms
,
testicular neoplasms
,
male genital neoplasms
,
gastrointestinal stromal tumors
,
hairy cell leukemia
,
vaginal neoplasms
,
spinal cord neoplasms
,
acinar cell carcinoma
,
adenoviridae infections
,
hypoxia
,
clear cell adenocarcinoma
,
adenomyoepithelioma
,
hereditary breast and ovarian cancer syndrome
,
basal cell neoplasms
,
urogenital neoplasms
,
male urogenital diseases
,
hemangiosarcoma
,
perivascular epithelioid cell neoplasms
,
neoplasms
,
carcinoma
,
lung neoplasms
,
breast neoplasms
,
colorectal neoplasms
,
triple negative breast neoplasms
,
squamous cell carcinoma
,
renal cell carcinoma
,
ovarian neoplasms
,
urinary bladder neoplasms
,
head and neck neoplasms
,
neoplasm metastasis
,
stomach neoplasms
,
adenocarcinoma
,
recurrence
,
prostatic neoplasms
,
liver neoplasms
,
hepatocellular carcinoma
,
esophageal neoplasms
,
endometrial neoplasms
,
uterine cervical neoplasms
,
transitional cell carcinoma
,
small cell lung carcinoma
,
hodgkin disease
,
urologic neoplasms
,
fallopian tube neoplasms
,
multiple myeloma
,
glioblastoma
,
ovarian epithelial carcinoma
,
squamous cell neoplasms
,
mesothelioma
,
biliary tract neoplasms
,
skin neoplasms
,
cholangiocarcinoma
,
esophageal squamous cell carcinoma
,
plasma cell neoplasms
,
malignant mesothelioma
,
peritoneal neoplasms
,
merkel cell carcinoma
,
cutaneous malignant melanoma
,
hematologic neoplasms
,
nasopharyngeal neoplasms
,
bile duct neoplasms
,
non-muscle invasive bladder neoplasms
,
gallbladder neoplasms
,
non-small-cell lung carcinoma
,
melanoma
,
squamous cell carcinoma of head and neck
Solumedrol
multiple sclerosis
,
lupus nephritis
,
systemic lupus erythematosus
,
neoplasms
,
hodgkin disease
,
inflammation
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