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Disease Reports

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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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

,

breast neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

myeloid leukemia acute

,

myeloid leukemia

,

,

,

neurofibromatosis 2

,

neurofibromatosis 1

,

neurofibromatoses

,

,

,

coronavirus infections

,

,

,

healthy volunteers/patients

,

,

,

,

neuroendocrine tumors

,

neoplasm metastasis

,

,

b-cell chronic lymphocytic leukemia

,

multiple hamartoma syndrome

,

carcinoid tumor

,

aplastic anemia

,

,

oligodendroglioma

,

,

,

,

cerebellar neoplasms

,

brain neoplasms

,

hepatocellular carcinoma

,

endometrial neoplasms

,

pancreatic neoplasms

,

neuroendocrine carcinoma

,

islet cell carcinoma

,

atopic dermatitis

,

,

,

hodgkin disease

,

,

rheumatoid arthritis

,

,

thrombocytopenic purpura idiopathic

,

thrombocytopenia

,

neurofibromatosis 2

,

neurofibromatosis 1

,

neurofibromatoses

,

,

t-cell lymphoma

,

t-cell lymphoma peripheral

,

t-cell lymphoma cutaneous

,

tuberous sclerosis

,

urinary bladder neoplasms

,

transitional cell carcinoma

,

,

,

myeloproliferative disorders

,

essential thrombocythemia

,

,

precursor t-cell lymphoblastic leukemia-lymphoma

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

non-hodgkin lymphoma

,

mantle-cell lymphoma

,

colorectal neoplasms

,

kidney neoplasms

,

,

,

multiple myeloma

,

plasma cell neoplasms

,

,

,

psoriatic arthritis

,

renal cell carcinoma

,

,

,

,

,

,

,

meningococcal meningitis

,

prostatic neoplasms

,

organ transplantation

,

graft rejection

,

pulmonary arterial hypertension

,

familial primary pulmonary hypertension

,

paroxysmal hemoglobinuria

,

,

chronic urticaria

,

,

hypersensitivity

,

food hypersensitivity

,

peanut hypersensitivity

,

juvenile arthritis

,

,

hereditary autoinflammatory diseases

,

,

familial mediterranean fever

,

,

,

,

,

,

attention deficit disorder with hyperactivity

,

,

,

major depressive disorder

,

depressive disorder

,

,

chronic hepatitis b

,

,

chronic hepatitis

,

heart transplantation

,

bone resorption

,

,

,

myeloid leukemia

,

kidney transplantation

,

breast neoplasms

,

,

bcr-abl positive chronic myelogenous leukemia

,

meningococcal infections

,

,

sickle cell anemia

,

liver transplantation

,

multiple sclerosis

,

chronic obstructive pulmonary disease

,

,

obstructive lung diseases

,

gastrointestinal stromal tumors

,

myeloid leukemia acute

,

human influenza

,

macular degeneration

,

lymphoid leukemia

,

,

,

alzheimer disease

,

choroidal neovascularization

,

pathologic neovascularization

,

,

beta-thalassemia

,

,

parkinson disease

,

relapsing-remitting multiple sclerosis

,

dry eye syndromes

,

type 2 diabetes mellitus

,

diabetes mellitus

,

polycythemia vera

,

primary myelofibrosis

,

graft vs host disease

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

myeloid leukemia chronic-phase

,

,

myelodysplastic syndromes

,

,

,

wet macular degeneration

,

,

,

cystic fibrosis

,

allergic bronchopulmonary aspergillosis

,

pulmonary aspergillosis

,

retinal detachment

,

vascular diseases

,

dissociative disorders

,

polypoidal choroidal vasculopathy

,

diabetic retinopathy

,

pneumococcal infections

,

hemochromatosis

,

,

philadelphia chromosome

,

,

stomach neoplasms

,

,

ankylosing spondylitis

,

spondylarthritis

,

hyperlipidemias

,

gastrointestinal stromal tumors

,

,

iron-deficiency anemia

,

iron deficiencies

,

,

,

,

small cell lung carcinoma

,

healthy volunteers/patients

,

,

primary myelofibrosis

,

myeloid leukemia acute

,

myeloid leukemia

,

,

hepatocellular carcinoma

,

,

plexiform neurofibroma

,

neurofibromatoses

,

,

,

,

chronic obstructive pulmonary disease

,

,

triple negative breast neoplasms

,

,

,

essential thrombocythemia

,

polycythemia vera

,

,

,

lupus nephritis

,

,

inflammatory breast neoplasms

,

prostatic neoplasms

,

precursor cell lymphoblastic leukemia-lymphoma

,

precursor t-cell lymphoblastic leukemia-lymphoma

,

b-cell chronic lymphocytic leukemia

,

lymphoid leukemia

,

,

wiskott-aldrich syndrome

,

bcr-abl positive chronic myelogenous leukemia

,

,

,

hyperlipoproteinemia type ii

,

organ transplantation

,

homozygous familial hypercholesterolemia

,

cryopyrin-associated periodic syndromes

,

myelodysplastic syndromes

,

,

liver transplantation

,

thrombocytopenic purpura idiopathic

,

thrombocytopenic purpura

,

,

parkinson disease

,

,

,

juvenile arthritis

,

,

,

,

hepatitis b virus

,

,

,

,

vision disorders

,

,

pituitary acth hypersecretion

,

acth-secreting pituitary adenoma

,

,

acute coronary syndrome

,

,

,

pulmonary eosinophilia

,

pancreatic neoplasms

,

,

,

kidney transplantation

,

,

hypercholesterolemia

,

cardiovascular diseases

,

atherosclerosis

,

chronic kidney failure

,

kidney diseases

,

thrombocytopenia

,

,

breast neoplasms

,

,

renal insufficiency

,

,

,

,

chronic renal insufficiency

,

,

renal replacement therapy

,

drug-related side effects and adverse reactions

,

heart transplantation

,

polycystic kidney diseases

,

autosomal dominant polycystic kidney

,

,

,

cystic fibrosis

,

pseudomonas infections

,

type 2 diabetes mellitus

,

diabetes mellitus

,

multiple myeloma

,

plasma cell neoplasms

,

transplantation

,

cytomegalovirus infections

,

communicable diseases

,

,

healthy volunteers/patients

,

hepatic insufficiency

,

esophageal neoplasms

,

,

,

small cell lung carcinoma

,

pancreatic ductal carcinoma

,

multiple myeloma

,

plasma cell neoplasms

,

bronchiolitis obliterans syndrome

,

congenital abnormalities

,

chromosome aberrations

,

brain stem neoplasms

,

chronic hepatitis c

,

chronic hepatitis

,

,

,

medulloblastoma

,

,

,

choroid plexus neoplasms

,

primitive neuroectodermal tumors

,

,

,

neuroepithelial neoplasms

,

ganglioneuroblastoma

,

neuroectodermal tumors

,

phenylketonurias

,

precursor t-cell lymphoblastic leukemia-lymphoma

,

nasopharyngeal carcinoma

,

ureteral neoplasms

,

pelvic neoplasms

,

adenoid cystic carcinoma

,

metabolic syndrome

,

follicular lymphoma

,

,

pancreatic neoplasms

,

kidney neoplasms

,

,

prostatic neoplasms

,

,

,

head and neck neoplasms

,

neurofibromatoses

,

,

liver neoplasms

,

,

,

neurofibromatosis 1

,

plexiform neurofibroma

,

systemic scleroderma

,

diffuse scleroderma

,

b-cell chronic lymphocytic leukemia

,

,

hematologic neoplasms

,

endometrial neoplasms

,

neuroendocrine carcinoma

,

central nervous system neoplasms

,

ovarian neoplasms

,

mantle-cell lymphoma

,

urinary bladder neoplasms

,

squamous cell carcinoma

,

uterine cervical neoplasms

,

myelodysplastic syndromes

,

non-hodgkin lymphoma

,

nervous system neoplasms

,

hypereosinophilic syndrome

,

systemic mastocytosis

,

,

uveal neoplasms

,

squamous cell carcinoma of head and neck

,

t-cell lymphoma

,

transitional cell carcinoma

,

primary myelofibrosis

,

,

graft vs host disease

,

adenocarcinoma of lung

,

diffuse intrinsic pontine glioma

,

disease progression

,

oligodendroglioma

,

neurofibromatosis 2

,

acoustic neuroma

,

,

hodgkin disease

,

islet cell carcinoma

,

hematologic diseases

,

myeloid leukemia accelerated phase

,

,

mast-cell leukemia

,

,

germ cell and embryonal neoplasms

,

,

anaplastic thyroid carcinoma

,

t-cell lymphoma peripheral

,

waldenstrom macroglobulinemia

,

castration-resistant prostatic neoplasms

,

,

cholangiocarcinoma

,

chronic disease

,

large-cell lymphoma anaplastic

,

plasma cell granuloma

,

,

,

,

,

gouty arthritis

,

endocrine gland neoplasms

,

,

urogenital neoplasms

,

male genital diseases

,

prostatic diseases

,

abdominal neoplasms

,

male genital neoplasms

,

genital diseases

,

sjogren's syndrome

,

retinitis pigmentosa

,

,

,

giant cell tumors

,

pigmented villonodular synovitis

,

giant cell tumor of tendon sheath

,

choroidal neovascularization

,

pathologic neovascularization

,

hemangioblastoma

,

von hippel-lindau disease

,

inflammatory breast neoplasms

,

invasive hydatidiform mole

,

multidrug-resistant tuberculosis

,

local neoplasm recurrence

,

t-cell lymphoma cutaneous

,

peutz-jeghers syndrome

,

acute coronary syndrome

,

st elevation myocardial infarction

,

malignant mesothelioma

,

,

type 1 diabetes mellitus

,

,

intestinal neoplasms

,

testicular neoplasms

,

,

brain stem infarctions

,

,

sturge-weber syndrome

,

klippel-trenaunay-weber syndrome

,

neurofibrosarcoma

,

nerve sheath neoplasms

,

carcinoma in situ

,

breast diseases

,

multiple hamartoma syndrome

,

,

urologic neoplasms

,

fallopian tube neoplasms

,

t-cell leukemia

,

localized scleroderma

,

myelomonocytic leukemia chronic

,

myelomonocytic leukemia acute

,

myelomonocytic leukemia juvenile

,

,

,

ovarian epithelial carcinoma

,

sickle cell anemia

,

streptococcus agalactiae

,

colorectal neoplasms

,

myeloid leukemia acute

,

,

,

neoplasm metastasis

,

brain neoplasms

,

hepatocellular carcinoma

,

,

thyroid neoplasms

,

thyroid diseases

,

chronic urticaria

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

,

triple negative breast neoplasms

,

,

colonic neoplasms

,

,

carcinoid tumor

,

pulmonary arterial hypertension

,

familial primary pulmonary hypertension

,

,

gastrointestinal neoplasms

,

stomach neoplasms

,

rectal neoplasms

,

,

meningococcal meningitis

,

meningococcal infections

,

rheumatoid arthritis

,

organ transplantation

,

graft rejection

,

digestive system neoplasms

,

cutaneous malignant melanoma

,

,

lymphangioleiomyomatosis

,

lupus nephritis

,

,

hypersensitivity

,

peanut hypersensitivity

,

multiple sclerosis

,

relapsing-remitting multiple sclerosis

,

interstitial lung diseases

,

,

lung transplantation

,

malignant carcinoid syndrome

,

serotonin syndrome

,

food hypersensitivity

,

psoriatic arthritis

,

,

muscular atrophy

,

spinal muscular atrophy

,

,

,

,

idiopathic pulmonary fibrosis

,

pulmonary fibrosis

,

,

chronic inducible urticaria

,

infectious skin diseases

,

bacterial skin diseases

,

,

,

myeloid leukemia

,

,

bcr-abl positive chronic myelogenous leukemia

,

breast neoplasms

,

,

,

non-small-cell lung carcinoma

,

renal cell carcinoma

,

,

kidney transplantation

,

philadelphia chromosome

,

lymphoid leukemia

,

neuroendocrine tumors

,

gastrointestinal stromal tumors

,

precursor cell lymphoblastic leukemia-lymphoma

,

,

myeloid leukemia chronic-phase

,

liver transplantation

,

renal insufficiency

,

tuberous sclerosis

,

chronic kidney failure

,

heart transplantation

,

kidney diseases

,

islet cell adenoma

,

,

,

macular degeneration

,

,

,

,

autosomal dominant polycystic kidney

,

communicable diseases

,

polycystic kidney diseases

,

,

diabetes mellitus

,

tick-borne encephalitis

,

,

immunosuppression therapy

,

cytomegalovirus infections

,

,

large cell carcinoma

,

vascular diseases

,

,

,

vision disorders

,

,

type 2 diabetes mellitus

,

cross-sectional studies

,

,

polyomavirus infections

,

cryopyrin-associated periodic syndromes

,

polycythemia vera

,

,

,

systolic heart failure

,

myocardial infarction

,

,

,

inferior wall myocardial infarction

,

friedreich ataxia

,

cerebellar ataxia

,

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

,

,

lymphoid leukemia

,

,

respiratory insufficiency

,

,

giant cell arteritis

,

polymyalgia rheumatica

,

lupus nephritis

,

,

multiple myeloma

,

plasma cell neoplasms

,

,

,

,

,

pulmonary sarcoidosis

,

b-cell chronic lymphocytic leukemia

,

,

healthy volunteers/patients

,

obstructive lung diseases

,

,

graft rejection

,

thrombocytopenic purpura idiopathic

,

thrombocytopenia

,

,

interstitial lung diseases

,

thrombocytopenic purpura

,

,

nephrotic syndrome

,

,

,

,

follicular lymphoma

,

kidney transplantation

,

,

chronic obstructive pulmonary disease

,

liver transplantation

,

atopic dermatitis

,

,

,

graft vs host disease

,

chronic kidney failure

,

,

cytomegalovirus infections

,

communicable diseases

,

kidney diseases

,

,

renal insufficiency

,

heart transplantation

,

multiple sclerosis

,

,

chronic renal insufficiency

,

,

renal replacement therapy

,

drug-related side effects and adverse reactions

,

,

relapsing-remitting multiple sclerosis

,

vascular diseases

,

,

cystic fibrosis

,

allergic bronchopulmonary aspergillosis

,

pulmonary aspergillosis

,

transplantation

,

,

end stage liver disease

,

,

disease progression

,

huntington disease

thoracic neoplasms

,

glandular and epithelial neoplasms

,

,

,

myelodysplastic syndromes

,

pharmacokinetics

,

soft tissue neoplasms

,

digestive system neoplasms

,

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

endocrine gland neoplasms

,

breast diseases

,

bronchial neoplasms

,

intraductal carcinoma noninfiltrating

,

,

,

pancreatic neoplasms

,

neoplasm metastasis

,

,

b-cell lymphoma

,

castration-resistant prostatic neoplasms

,

large b-cell lymphoma diffuse

,

brain neoplasms

,

non-hodgkin lymphoma

,

t-cell lymphoma

,

,

neuroendocrine tumors

,

cholangiocarcinoma

,

follicular lymphoma

,

myeloid leukemia acute

,

neuroendocrine carcinoma

,

pancreatic ductal carcinoma

,

kidney neoplasms

,

thyroid neoplasms

,

t-cell lymphoma peripheral

,

uterine neoplasms

,

myeloid leukemia

,

uveal neoplasms

,

lymphoid leukemia

,

,

invasive hydatidiform mole

,

female genital neoplasms

,

small cell carcinoma

,

urethral neoplasms

,

ureteral neoplasms

,

colonic neoplasms

,

vulvar neoplasms

,

b-cell lymphoma marginal zone

,

,

thyroid diseases

,

,

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

,

,

,

mullerian mixed tumor

,

,

cutaneous malignant melanoma

,

thymus neoplasms

,

rectal neoplasms

,

hypopharyngeal neoplasms

,

male genital neoplasms

,

bile duct neoplasms

,

respiratory tract infections

,

papillomavirus infections

,

,

adenoid cystic carcinoma

,

,

male breast neoplasms

,

,

malignant fibrous histiocytoma

,

epstein-barr virus infections

,

adenocarcinoma of lung

,

,

neurofibrosarcoma

,

nerve sheath neoplasms

,

,

sezary syndrome

,

mycosis fungoides

,

t-cell lymphoma cutaneous

,

,

pelvic neoplasms

,

hemangiosarcoma

,

,

germ cell and embryonal neoplasms

,

neoplasms by histologic type

,

adenoviridae infections

,

basal cell carcinoma

,

anaplastic thyroid carcinoma

,

reactive arthritis

,

clear cell adenocarcinoma

,

gestational trophoblastic disease

,

alveolar soft part sarcoma

,

precursor cell lymphoblastic leukemia-lymphoma

,

biphenotypic leukemia acute

,

local neoplasm recurrence

,

,

,

meningeal carcinomatosis

,

papillomaviridae

,

immunoblastic lymphadenopathy

,

enteropathy-associated t-cell lymphoma

,

intestinal diseases

,

,

carcinoma in situ

,

cervical intraepithelial neoplasia

,

uterine cervical dysplasia

,

squamous intraepithelial lesions of the cervix

,

central nervous system diseases

,

extranodal nk-t-cell lymphoma

,

,

,

,

unknown primary neoplasms

,

synovial sarcoma

,

,

oral leukoplakia

,

,

lymphoproliferative disorders

,

,

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

,

gallbladder neoplasms

,

acinar cell carcinoma

,

adenomyoepithelioma

,

hereditary breast and ovarian cancer syndrome

,

maxillary sinus neoplasms

,

basal cell neoplasms

,

urogenital neoplasms

,

male urogenital diseases

,

urogenital diseases

,

adenosquamous carcinoma

,

,

,

breast neoplasms

,

colorectal neoplasms

,

,

triple negative breast neoplasms

,

renal cell carcinoma

,

ovarian neoplasms

,

,

,

urinary bladder neoplasms

,

head and neck neoplasms

,

prostatic neoplasms

,

esophageal neoplasms

,

stomach neoplasms

,

liver neoplasms

,

endometrial neoplasms

,

hepatocellular carcinoma

,

uterine cervical neoplasms

,

ovarian epithelial carcinoma

,

small cell lung carcinoma

,

transitional cell carcinoma

,

hodgkin disease

,

fallopian tube neoplasms

,

urologic neoplasms

,

multiple myeloma

,

,

squamous cell neoplasms

,

,

plasma cell neoplasms

,

,

esophageal squamous cell carcinoma

,

malignant mesothelioma

,

biliary tract neoplasms

,

peritoneal neoplasms

,

microsatellite instability

,

merkel cell carcinoma

,

non-muscle invasive bladder neoplasms

,

hematologic neoplasms

,

hypersensitivity

,

nasopharyngeal neoplasms

,

nasopharyngeal carcinoma

,

disease progression

,

muscle neoplasms

,

,

non-small-cell lung carcinoma

,

,

squamous cell carcinoma of head and neck

,

squamous cell carcinoma

giant cell arteritis

,

polymyalgia rheumatica

,

,

lymphoid leukemia

,

,

b-cell chronic lymphocytic leukemia

,

breast neoplasms

,

,

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

,

graft vs host disease

,

bronchiolitis obliterans syndrome

,

lupus nephritis

,

,

,

peritoneal neoplasms

,

intestinal obstruction

,

,

intermediate uveitis

,

,

follicular lymphoma

,

prostatic neoplasms

,

kidney transplantation

,

liver transplantation

,

chronic kidney failure

,

,

,

healthy volunteers/patients

,

chronic renal insufficiency

,

kidney diseases

,

,

renal replacement therapy

,

renal insufficiency

,

liver cirrhosis

,

drug-related side effects and adverse reactions

,

cytomegalovirus infections

,

communicable diseases

,

multiple sclerosis

,

,

relapsing-remitting multiple sclerosis

,

,

heart transplantation

,

,

,

cataract extraction

,

capsule opacification

,

medical sociology

,

castration-resistant prostatic neoplasms

,

,

neoplasm metastasis

,

bone marrow diseases

,

non-small-cell lung carcinoma

,

,

squamous cell neoplasms

,

,

prostatic neoplasms

,

hypersensitivity

,

,

transitional cell carcinoma

,

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