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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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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

,

therapeutic equivalency

pancreatic neoplasms

,

inflammatory bowel diseases

,

waldenstrom macroglobulinemia

,

myeloid leukemia acute

,

myelodysplastic syndromes

,

renal insufficiency

,

brain neoplasms

,

glandular and epithelial neoplasms

,

,

delayed graft function

,

hypersensitivity

,

venous thromboembolism

,

thromboembolism

,

pharmacokinetics

,

,

central nervous system diseases

,

,

,

endometrial neoplasms

,

,

mullerian mixed tumor

,

,

,

breast neoplasms

,

rheumatoid arthritis

,

,

b-cell chronic lymphocytic leukemia

,

neoplasm metastasis

,

,

non-hodgkin lymphoma

,

,

colorectal neoplasms

,

ulcerative colitis

,

,

,

,

triple negative breast neoplasms

,

small cell lung carcinoma

,

autoimmune diseases

,

,

kidney neoplasms

,

,

female genital neoplasms

,

biliary tract neoplasms

,

neuroendocrine tumors

,

,

kidney transplantation

,

nephrotic syndrome

,

focal segmental glomerulosclerosis

,

,

lipoid nephrosis

,

discoid lupus erythematosus

,

cutaneous lupus erythematosus

,

lupus nephritis

,

,

,

diabetic nephropathies

,

kidney diseases

,

atopic dermatitis

,

,

,

alopecia areata

,

,

hematologic neoplasms

,

gastrointestinal neoplasms

,

esophageal neoplasms

,

pancreatic ductal carcinoma

,

hidradenitis suppurativa

,

,

polycythemia vera

,

,

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

gastrointestinal stromal tumors

,

,

alveolar soft part sarcoma

,

solitary fibrous tumors

,

,

rhabdomyosarcoma

,

primitive neuroectodermal tumors peripheral

,

neurofibrosarcoma

,

nerve sheath neoplasms

,

giant cell tumor of bone

,

giant cell tumors

,

hemangiopericytoma

,

adenocarcinoma of lung

,

merkel cell carcinoma

,

castration-resistant prostatic neoplasms

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

,

non-small-cell lung carcinoma

,

,

renal cell carcinoma

,

systemic lupus erythematosus

,

lymphoid leukemia

,

multiple myeloma

,

philadelphia chromosome

,

prostatic neoplasms

,

,

plasma cell neoplasms

,

precursor cell lymphoblastic leukemia-lymphoma

,

psoriatic arthritis

,

hepatocellular carcinoma

,

liver neoplasms

,

,

head and neck neoplasms

,

squamous cell carcinoma of head and neck

,

,

myeloid leukemia accelerated phase

,

sjogren's syndrome

,

squamous cell carcinoma

,

rheumatic diseases

,

,

bcr-abl positive chronic myelogenous leukemia

,

myeloid leukemia

,

healthy volunteers/patients

,

,

myeloid leukemia chronic-phase

,

coronary artery disease

,

pleural effusion

,

,

,

cardiomyopathies

,

dilated cardiomyopathy

,

alzheimer disease

,

progressive supranuclear palsy

,

,

,

urinary bladder neoplasms

,

transitional cell carcinoma

,

noise-induced hearing loss

,

,

parkinson disease

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

vasoconstriction

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

,

,

cholangiocarcinoma

,

neuroendocrine tumors

,

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

,

second primary neoplasms

,

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

,

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

,

,

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

atrial fibrillation

,

chronic kidney failure

,

renal insufficiency

,

kidney transplantation

,

hyperparathyroidism

,

secondary hyperparathyroidism

colonic neoplasms

,

non-hodgkin lymphoma

,

pharmacokinetics

,

hematologic neoplasms

,

b-cell chronic lymphocytic leukemia

,

neoplasm micrometastasis

,

neuroendocrine tumors

,

medical sociology

,

thrombocytopenia

,

connective and soft tissue neoplasms

,

paraneoplastic syndromes

,

,

renal insufficiency

,

vaginal neoplasms

,

vulvar neoplasms

,

rectal neoplasms

,

,

,

,

colorectal neoplasms

,

,

kidney neoplasms

,

liver neoplasms

,

,

biliary tract neoplasms

,

,

,

hepatocellular carcinoma

,

thyroid neoplasms

,

,

,

gallbladder neoplasms

,

cholangiocarcinoma

,

pleural effusion

,

malignant pleural effusion

,

,

,

adenosquamous carcinoma

,

gastrointestinal stromal tumors

,

hemangiosarcoma

,

hyperthermic intraperitoneal chemotherapy

,

synovial sarcoma

,

urogenital neoplasms

,

thymus neoplasms

,

small cell carcinoma

,

dna repair-deficiency disorders

,

thyroid diseases

,

uterine neoplasms

,

myeloid leukemia acute

,

,

,

carcinoma in situ

,

,

,

germ cell and embryonal neoplasms

,

,

large cell carcinoma

,

,

papillomaviridae

,

neuroendocrine carcinoma

,

residual neoplasm

,

,

,

cognitive dysfunction

,

myeloproliferative disorders

,

multiple myeloma

,

alopecia areata

,

,

lacrimal apparatus diseases

,

adrenocortical carcinoma

,

,

heavy ion radiotherapy

,

merkel cell carcinoma

,

microsatellite instability

,

breast carcinoma in situ

,

lobular carcinoma

,

testicular neoplasms

,

branchio-oto-renal syndrome

,

,

,

,

left ventricular dysfunction

,

ventricular dysfunction

,

,

,

somatoform disorders

,

disseminated intravascular coagulation

,

tonsillar neoplasms

,

glandular and epithelial neoplasms

,

,

cardiovascular diseases

,

,

intraductal carcinoma noninfiltrating

,

anaplastic thyroid carcinoma

,

antineoplastic combined chemotherapy protocols

,

,

urologic diseases

,

urinary bladder diseases

,

,

papillomavirus infections

,

neurofibrosarcoma

,

nerve sheath neoplasms

,

lymphatic metastasis

,

,

bone marrow diseases

,

epstein-barr virus infections

,

,

hormone-dependent neoplasms

,

ampulla of vater cancer

,

gastrointestinal diseases

,

digestive system diseases

,

mycobacterium infections

,

mediastinal neoplasms

,

palliative care

,

mullerian mixed tumor

,

pancoast syndrome

,

penile neoplasms

,

thromboembolism

,

intestinal neoplasms

,

myelodysplastic syndromes

,

precancerous conditions

,

plasma cell neoplasms

,

,

,

,

central nervous system neoplasms

,

nervous system neoplasms

,

adenoid cystic carcinoma

,

intestinal obstruction

,

alphapapillomavirus

,

carcinoid tumor

,

,

pancreatic ductal carcinoma

,

hodgkin disease

,

maxillary sinus neoplasms

,

adolescent development

,

,

cancer vaccines

,

radiation effects

,

perioperative period

,

ovarian neoplasms

,

nasopharyngeal carcinoma

,

,

nasopharyngeal neoplasms

,

,

ovarian epithelial carcinoma

,

pancreatic neoplasms

,

urinary bladder neoplasms

,

esophageal squamous cell carcinoma

,

urologic neoplasms

,

transitional cell carcinoma

,

fallopian tube neoplasms

,

hypersensitivity

,

uterine cervical neoplasms

,

peritoneal neoplasms

,

small cell lung carcinoma

,

laryngeal neoplasms

,

endometrial neoplasms

,

oropharyngeal neoplasms

,

,

renal cell carcinoma

,

hypopharyngeal neoplasms

,

inflammatory breast neoplasms

,

salivary gland neoplasms

,

mouth neoplasms

,

,

,

chemotherapy-induced febrile neutropenia

,

disease progression

,

neoadjuvant therapy

,

non-muscle invasive bladder neoplasms

,

invasive hydatidiform mole

,

laryngeal diseases

,

drug-related side effects and adverse reactions

,

gastrointestinal neoplasms

,

,

,

unknown primary neoplasms

,

ureteral neoplasms

,

malignant fibrous histiocytoma

,

,

tongue neoplasms

,

digestive system neoplasms

,

urethral neoplasms

,

brain stem neoplasms

,

paranasal sinus neoplasms

,

circulating neoplastic cells

,

endometrioid carcinoma

,

breast diseases

,

bronchiolo-alveolar adenocarcinoma

,

hereditary breast and ovarian cancer syndrome

,

disease-free survival

,

verrucous carcinoma

,

brain neoplasms

,

cystadenocarcinoma

,

chemoradiotherapy

,

rhabdomyosarcoma

,

,

mucinous cystadenocarcinoma

,

mucinous adenocarcinoma

,

serous cystadenocarcinoma

,

clear cell adenocarcinoma

,

neoplasms by site

,

local neoplasm recurrence

,

ductal carcinoma breast

,

bronchogenic carcinoma

,

ductal carcinoma

,

neurotoxicity syndromes

,

healthy volunteers/patients

,

,

progression-free survival

,

postoperative complications

,

,

acinar cell carcinoma

,

,

pelvic neoplasms

,

induction chemotherapy

,

respiratory tract diseases

,

,

respiratory tract neoplasms

,

thoracic neoplasms

,

bronchial neoplasms

,

genetic predisposition to disease

,

disease susceptibility

,

,

pharyngeal neoplasms

,

wasting syndrome

,

,

,

therapeutic equivalency

,

adenomyoepithelioma

,

,

directly observed therapy

,

alveolar rhabdomyosarcoma

,

embryonal rhabdomyosarcoma

,

breast neoplasms

,

non-small-cell lung carcinoma

,

,

prostatic neoplasms

,

,

,

stomach neoplasms

,

,

squamous cell carcinoma

,

esophageal neoplasms

,

squamous cell carcinoma of head and neck

,

head and neck neoplasms

,

castration-resistant prostatic neoplasms

,

triple negative breast neoplasms

,

neoplasm metastasis

,

squamous cell neoplasms

,

,

adenocarcinoma of lung

,

male breast neoplasms

,

peripheral nervous system diseases

,

febrile neutropenia

,

,

second primary neoplasms

,

wounds and injuries

,

chemical and drug induced liver injury

,

meningeal carcinomatosis

,

,

,

,

,

,

breast cancer lymphedema

,

taste disorders

,

,

,

,

,

prospective studies

,

pharmacogenomic testing

,

,

chemotherapy-related cognitive impairment

,

,

,

,

postoperative pain

,

,

,

,

pathologic complete response

,

hand-foot syndrome

,

intravesical administration

,

mycobacterium bovis

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