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Company ReportsPTC 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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Clinical Trials

Financial

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Company Revenue

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Revenue by Drug

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

healthy volunteers/patients

,

renal insufficiency

,

,

hepatic insufficiency

,

muscular dystrophies

,

duchenne muscular dystrophy

,

limb-girdle muscular dystrophies

ELADOCAGENE EXUPARVOVEC

argininosuccinic aciduria

,

phenylketonurias

,

breast neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

myeloid leukemia acute

,

myeloid leukemia

,

,

,

neurofibromatosis 2

,

neurofibromatosis 1

,

neurofibromatoses

,

,

,

coronavirus infections

,

,

,

,

acquired immunodeficiency syndrome

,

,

immunologic deficiency syndromes

,

colorectal neoplasms

,

urinary bladder neoplasms

,

,

non-small-cell lung carcinoma

,

pancreatic neoplasms

,

neoplasm metastasis

,

squamous cell carcinoma of head and neck

,

,

squamous cell carcinoma

,

esophageal squamous cell carcinoma

stomach neoplasms

,

healthy volunteers/patients

,

alzheimer disease

,

female genital neoplasms

,

,

chronic hepatitis b

,

,

major depressive disorder

,

depressive disorder

,

triple negative breast neoplasms

,

pancreatic neoplasms

,

coronary disease

,

coronary artery disease

,

myocardial ischemia

,

,

,

sickle cell anemia

,

,

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

parkinson disease

,

autistic disorder

,

autism spectrum disorder

,

pervasive child development disorders

,

chronic hepatitis

,

rectal neoplasms

,

,

,

,

b-cell chronic lymphocytic leukemia

,

lymphoid leukemia

,

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

stomach neoplasms

,

esophageal neoplasms

,

,

neoplasm metastasis

,

antiphospholipid syndrome

,

respiratory insufficiency

,

,

colorectal neoplasms

,

non-small-cell lung carcinoma

,

lupus nephritis

,

human influenza

,

breast neoplasms

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

non-hodgkin lymphoma

,

myeloid leukemia acute

,

multiple sclerosis

,

,

hepatocellular carcinoma

,

colonic neoplasms

,

thrombocytopenic purpura idiopathic

,

liver cirrhosis

,

neuromyelitis optica

,

membranous glomerulonephritis

,

chronic progressive multiple sclerosis

,

relapsing-remitting multiple sclerosis

,

nephrotic syndrome

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

hemolytic anemia autoimmune

,

,

chronic hepatitis c

,

,

,

rheumatoid arthritis

,

,

,

juvenile arthritis

,

,

follicular lymphoma

,

,

,

myelodysplastic syndromes

,

,

,

acquired immunodeficiency syndrome

,

kidney transplantation

,

drug-related side effects and adverse reactions

,

heart disease risk factors

,

cognitive dysfunction

healthy volunteers/patients

,

,

bcr-abl positive chronic myelogenous leukemia

,

type 2 diabetes mellitus

,

glandular and epithelial neoplasms

,

,

gata2 deficiency

,

,

fallopian tube diseases

,

neurologic manifestations

,

,

bronchiolitis obliterans syndrome

,

interdigitating dendritic cell sarcoma

,

myeloproliferative disorders

,

neoplasms by histologic type

,

adenosquamous carcinoma

,

chronic hepatitis c

,

,

venous thromboembolism

,

thromboembolism

,

pharmacokinetics

,

central nervous system neoplasms

,

,

behcet syndrome

,

aids-related complex

,

pleural effusion

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

thoracic neoplasms

,

malignant pleural effusion

,

pleural diseases

,

pleural neoplasms

,

immunosuppression therapy

,

small cell lung carcinoma

,

transitional cell carcinoma

,

ovarian neoplasms

,

non-hodgkin lymphoma

,

triple negative breast neoplasms

,

squamous cell carcinoma

,

urologic neoplasms

,

,

biliary tract neoplasms

,

malignant mesothelioma

,

large b-cell lymphoma diffuse

,

,

uterine cervical neoplasms

,

neoplasms by site

,

castration-resistant prostatic neoplasms

,

endometrial neoplasms

,

,

squamous cell neoplasms

,

b-cell lymphoma

,

,

uveal neoplasms

,

,

hematologic neoplasms

,

t-cell lymphoma peripheral

,

t-cell lymphoma

,

b-cell chronic lymphocytic leukemia

,

,

neuroendocrine tumors

,

gastrointestinal neoplasms

,

adenocarcinoma of lung

,

myeloid leukemia acute

,

cholangiocarcinoma

,

graft vs host disease

,

interstitial lung diseases

,

follicular lymphoma

,

merkel cell carcinoma

,

peritoneal neoplasms

,

spinocerebellar degenerations

,

,

graft rejection

,

psychology rejection

,

clear cell adenocarcinoma

,

,

,

,

rhabdomyosarcoma

,

,

autoimmune diseases

,

multiple sclerosis

,

waldenstrom macroglobulinemia

,

extranodal nk-t-cell lymphoma

,

precursor cell lymphoblastic leukemia-lymphoma

,

burkitt lymphoma

,

female genital neoplasms

,

endometrioid carcinoma

,

,

,

urogenital neoplasms

,

organ transplantation

,

neuroendocrine carcinoma

,

urinary bladder diseases

,

microsatellite instability

,

basal cell carcinoma

,

basal cell neoplasms

,

diffuse scleroderma

,

laryngeal diseases

,

,

heart transplantation

,

,

male breast neoplasms

,

medulloblastoma

,

,

b-cell leukemia

,

tertiary lymphoid structures

,

oropharyngeal neoplasms

,

,

prostatic diseases

,

non-muscle invasive bladder neoplasms

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

diffuse intrinsic pontine glioma

,

diabetic neuropathies

,

nephrotic syndrome

,

focal segmental glomerulosclerosis

,

lipoid nephrosis

,

diabetic nephropathies

,

kidney diseases

,

common variable immunodeficiency

,

large-cell lymphoma anaplastic

,

,

phenylketonurias

,

bile duct neoplasms

,

serous cystadenocarcinoma

,

papillary thyroid cancer

,

,

salivary gland neoplasms

,

genetic therapy

,

smoldering multiple myeloma

,

uterine neoplasms

,

large cell carcinoma

,

endometrial stromal tumors

,

small cell carcinoma

,

penile neoplasms

,

adrenocortical carcinoma

,

digestive system diseases

,

,

,

,

,

invasive hydatidiform mole

,

,

,

,

bronchiolo-alveolar adenocarcinoma

,

drug-related side effects and adverse reactions

,

carcinoid tumor

,

hypersensitivity

,

thyroid neoplasms

,

precancerous conditions

,

bronchopulmonary dysplasia

,

tobacco smoking

,

b-cell lymphoma marginal zone

,

mantle-cell lymphoma

,

meningeal carcinomatosis

,

systemic scleroderma

,

patient preference

,

,

oral leukoplakia

,

churg-strauss syndrome

,

microscopic polyangiitis

,

oligodendroglioma

,

myeloid leukemia

,

,

,

ankylosing spondylitis

,

,

inflammatory breast neoplasms

,

immunoglobulin g4-related disease

,

,

,

progressive multifocal leukoencephalopathy

,

gastrointestinal stromal tumors

,

male urogenital diseases

,

female urogenital diseases

,

female urogenital diseases and pregnancy complications

,

urogenital diseases

,

reactive arthritis

,

hereditary nonpolyposis colorectal neoplasms

,

,

aplastic anemia

,

,

,

non-small-cell lung carcinoma

,

colorectal neoplasms

,

neoplasm metastasis

,

squamous cell carcinoma of head and neck

,

prostatic neoplasms

,

head and neck neoplasms

,

breast neoplasms

,

pancreatic neoplasms

,

liver neoplasms

,

hepatocellular carcinoma

,

urinary bladder neoplasms

,

,

brain neoplasms

,

stomach neoplasms

,

hodgkin disease

,

multiple myeloma

,

colonic neoplasms

,

,

,

rectal neoplasms

,

esophageal neoplasms

,

,

,

,

cutaneous malignant melanoma

,

pancreatic ductal carcinoma

,

fallopian tube neoplasms

,

systemic lupus erythematosus

,

myelodysplastic syndromes

,

plasma cell neoplasms

,

,

ovarian epithelial carcinoma

,

,

,

esophageal squamous cell carcinoma

,

ulcerative colitis

,

juvenile arthritis

,

psoriatic arthritis

,

sjogren's syndrome

,

,

,

lupus nephritis

,

giant cell arteritis

,

respiratory tract diseases

,

granulomatosis with polyangiitis

,

anti-neutrophil cytoplasmic antibody-associated vasculitis

,

hyperlipidemias

,

polymyalgia rheumatica

,

vascular diseases

,

,

systemic vasculitis

,

disease progression

,

pathologic complete response

,

,

rheumatoid arthritis

,

renal cell carcinoma

,

,

,

kidney transplantation

,

acquired immunodeficiency syndrome

,

,

kidney neoplasms

,

delayed graft function

,

chronic kidney failure

,

coronary artery disease

,

,

hiv-associated lipodystrophy syndrome

,

epstein-barr virus infections

,

,

transplantation

,

mitochondrial diseases

,

,

,

parkinson disease

,

,

cardiomyopathies

,

dilated cardiomyopathy

,

,

,

alzheimer disease

,

progressive supranuclear palsy

,

,

,

diabetes mellitus

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

Bos taurus adrenal cortex corticosteroids

,

atrial fibrillation

,

,

coronary artery disease

,

,

chronic renal insufficiency

,

chronic kidney failure

,

kidney transplantation

,

pulmonary fibrosis

,

secondary hyperparathyroidism

uveal neoplasms

,

,

,

multiple sclerosis

,

lupus nephritis

,

systemic lupus erythematosus

,

,

hodgkin disease

,

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