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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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Annual

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Projections

*Quarterly sales USD (in millions)

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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

,

,

urologic neoplasms

,

urethral neoplasms

,

transitional cell carcinoma

,

ureteral neoplasms

,

pelvic neoplasms

,

prostatic neoplasms

,

urinary bladder neoplasms

,

acquired immunodeficiency syndrome

,

chronic hepatitis c

,

drug-related side effects and adverse reactions

,

,

myeloid leukemia

,

ulcerative colitis

,

,

,

rheumatoid arthritis

,

myeloid leukemia acute

,

atopic dermatitis

,

hidradenitis suppurativa

,

,

,

,

pathologic constriction

,

inflammatory bowel diseases

,

intestinal diseases

healthy volunteers/patients

,

,

knee osteoarthritis

,

,

,

,

telomeric repeat binding protein 2

,

plexiform neurofibroma

,

coronary disease

,

coronary artery disease

,

myocardial ischemia

,

hypersensitivity

,

urologic neoplasms

,

uveal neoplasms

,

,

rectal neoplasms

,

,

esophageal neoplasms

,

squamous cell carcinoma

,

rheumatoid arthritis

,

endometrial neoplasms

,

pancreatic neoplasms

,

urinary bladder neoplasms

,

fallopian tube neoplasms

,

neurofibromatosis 1

,

neurofibromatoses

,

multiple myeloma

,

pancreatic ductal carcinoma

,

small cell carcinoma

,

oropharyngeal neoplasms

,

serous cystadenocarcinoma

,

hereditary breast and ovarian cancer syndrome

,

type 1 diabetes mellitus

,

transitional cell carcinoma

,

hematologic neoplasms

,

,

uterine neoplasms

,

adenocarcinoma of lung

,

waldenstrom macroglobulinemia

,

,

esophageal squamous cell carcinoma

,

mouth neoplasms

,

squamous cell neoplasms

,

,

laryngeal neoplasms

,

,

diabetic neuropathies

,

b-cell lymphoma marginal zone

,

neurofibrosarcoma

,

nerve sheath neoplasms

,

chronic obstructive pulmonary disease

,

parkinson disease

,

hereditary sensory and autonomic neuropathies

,

urethral neoplasms

,

ureteral neoplasms

,

pelvic neoplasms

,

castration-resistant prostatic neoplasms

,

precursor cell lymphoblastic leukemia-lymphoma

,

,

mullerian mixed tumor

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

epstein-barr virus infections

,

,

,

peripheral nervous system diseases

,

,

uterine cervical neoplasms

,

residual neoplasm

,

pleural effusion

,

malignant pleural effusion

,

myocardial infarction

,

reperfusion injury

,

breast neoplasms

,

,

,

ovarian neoplasms

,

colorectal neoplasms

,

neoplasm metastasis

,

hepatocellular carcinoma

,

liver neoplasms

,

,

small cell lung carcinoma

,

,

large b-cell lymphoma diffuse

,

biliary tract neoplasms

,

non-hodgkin lymphoma

,

stomach neoplasms

,

squamous cell carcinoma of head and neck

,

,

chronic renal insufficiency

,

mantle-cell lymphoma

,

b-cell lymphoma

,

head and neck neoplasms

,

,

triple negative breast neoplasms

,

lymphoid leukemia

,

,

,

,

malignant mesothelioma

,

,

,

cardiovascular diseases

,

chronic kidney failure

,

kidney neoplasms

,

laryngeal diseases

,

communicable diseases

,

infectious skin diseases

,

,

breast diseases

,

,

,

ovarian reserve

,

respiratory syncytial virus infections

,

,

inflammatory bowel diseases

,

pituitary dwarfism

,

hypopituitarism

,

,

gastrointestinal stromal tumors

,

lymphoproliferative disorders

,

unknown primary neoplasms

,

postpartum hemorrhage

,

acute coronary syndrome

,

,

male breast neoplasms

,

renal cell carcinoma

,

type 2 diabetes mellitus

,

non-small-cell lung carcinoma

,

diabetes mellitus

,

,

prostatic neoplasms

,

b-cell chronic lymphocytic leukemia

,

,

insulin resistance

,

ovarian epithelial carcinoma

,

kidney diseases

,

,

,

,

,

lymphatic diseases

,

b-cell leukemia

,

left ventricular dysfunction

,

ventricular dysfunction

,

,

,

,

,

bacterial pneumonia

,

,

,

,

,

family relations

,

,

polyneuropathies

,

familial amyloid neuropathies

,

amyloid neuropathies

,

familial amyloidosis

,

respiratory syncytial viruses

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

Bos taurus adrenal cortex corticosteroids

precursor cell lymphoblastic leukemia-lymphoma

,

lymphoid leukemia

,

,

,

,

respiratory insufficiency

,

,

giant cell arteritis

,

polymyalgia rheumatica

,

lupus nephritis

,

multiple myeloma

,

breast neoplasms

,

,

,

animal mammary neoplasms

,

ornithine carbamoyltransferase deficiency disease

,

,

,

,

,

pulmonary sarcoidosis

,

b-cell chronic lymphocytic leukemia

,

hypersensitivity

,

,

healthy volunteers/patients

,

,

,

systemic lupus erythematosus

,

prostatic neoplasms

,

urogenital neoplasms

,

male genital neoplasms

,

hematopoietic stem cell transplantation

,

bronchiolitis obliterans syndrome

,

,

granulomatosis with polyangiitis

,

anti-neutrophil cytoplasmic antibody-associated vasculitis

,

microscopic polyangiitis

,

obstructive lung diseases

,

,

graft rejection

,

psychology rejection

,

thrombocytopenic purpura idiopathic

,

thrombocytopenia

,

,

,

,

nephrotic syndrome

,

,

,

,

follicular lymphoma

,

kidney transplantation

,

,

chronic obstructive pulmonary disease

,

graft vs host disease

,

liver transplantation

,

atopic dermatitis

,

,

,

renal insufficiency

,

chronic kidney failure

,

kidney diseases

,

,

cytomegalovirus infections

,

communicable diseases

,

heart transplantation

,

multiple sclerosis

,

,

chronic renal insufficiency

,

,

renal replacement therapy

,

drug-related side effects and adverse reactions

,

,

relapsing-remitting multiple sclerosis

,

cystic fibrosis

,

allergic bronchopulmonary aspergillosis

,

transplantation

,

end stage liver disease

,

,

disease progression

,

squamous cell carcinoma of head and neck

,

,

,

triple negative breast neoplasms

,

renal cell carcinoma

,

,

non-hodgkin lymphoma

,

multiple myeloma

,

urologic neoplasms

,

urinary bladder neoplasms

,

transitional cell carcinoma

,

colorectal neoplasms

,

prostatic neoplasms

,

non-small-cell lung carcinoma

,

,

hepatocellular carcinoma

,

liver neoplasms

,

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