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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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Revenue by Drug

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Marketed Drugs

Therapeutic Areas:

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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

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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

,

friedreich ataxia

,

cerebellar ataxia

,

mitochondrial diseases

,

,

,

,

,

diffuse cerebral sclerosis of schilder

,

drug resistant epilepsy

,

,

breast neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

myeloid leukemia acute

,

myeloid leukemia

,

,

,

neurofibromatosis 2

,

neurofibromatosis 1

,

neurofibromatoses

,

,

,

coronavirus infections

,

,

endometrial neoplasms

,

,

,

,

myeloid leukemia acute

,

myeloid leukemia

,

deficiency diseases

,

paraproteinemias

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

lymphoid leukemia

,

breast neoplasms

,

immune system diseases

,

hematologic neoplasms

,

type 2 diabetes mellitus

,

,

metabolic diseases

,

,

,

immunoglobulin g4-related disease

,

,

falciparum malaria

,

,

,

,

,

polycystic kidney diseases

,

autosomal dominant polycystic kidney

,

,

renal insufficiency

,

hematologic diseases

,

blood protein disorders

,

neoplasms by histologic type

,

prostatic neoplasms

,

circulating neoplastic cells

,

heart transplantation

,

non-small-cell lung carcinoma

,

,

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

esophageal neoplasms

,

,

neoplasm metastasis

,

,

aplastic anemia

,

multiple myeloma

,

plasma cell neoplasms

,

hypercholesterolemia

,

hyperlipoproteinemia type ii

,

meningococcal infections

,

hemolytic anemia autoimmune

,

,

meningococcal meningitis

,

stomach neoplasms

,

,

kidney diseases

,

gaucher disease

,

smoldering multiple myeloma

,

,

chronic renal insufficiency

,

hyperphosphatemia

,

multiple sclerosis

,

,

homozygous familial hypercholesterolemia

,

,

,

healthy volunteers/patients

,

,

glycogen storage disease type ii

,

glycogen storage disease

,

atopic dermatitis

,

,

,

,

diabetes mellitus

,

,

glycogen storage disease type iii

,

non-hodgkin lymphoma

,

,

postmenopausal osteoporosis

,

lymphoproliferative disorders

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

,

chronic urticaria

,

,

brain neoplasms

,

hepatocellular carcinoma

,

,

thyroid neoplasms

,

thyroid diseases

,

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

,

multiple sclerosis

,

relapsing-remitting multiple sclerosis

,

organ transplantation

,

graft rejection

,

digestive system neoplasms

,

cutaneous malignant melanoma

,

,

lymphangioleiomyomatosis

,

lupus nephritis

,

,

hypersensitivity

,

peanut hypersensitivity

,

chronic inducible urticaria

,

hidradenitis suppurativa

,

,

interstitial lung diseases

,

,

lung transplantation

,

malignant carcinoid syndrome

,

serotonin syndrome

,

food hypersensitivity

,

psoriatic arthritis

,

,

muscular atrophy

,

spinal muscular atrophy

,

,

,

,

idiopathic pulmonary fibrosis

,

pulmonary fibrosis

,

,

infectious skin diseases

,

bacterial skin diseases

,

,

myasthenia gravis

,

muscle weakness

,

,

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

,

open-angle glaucoma

,

alzheimer disease

,

,

myocardial infarction

,

,

,

renal cell carcinoma

,

ovarian neoplasms

,

urinary bladder neoplasms

,

fallopian tube neoplasms

,

,

ovarian epithelial carcinoma

,

,

,

barrett esophagus

,

chronic progressive multiple sclerosis

,

vitamin d deficiency

,

hypersensitivity

,

oral lichen planus

,

,

epidermolysis bullosa

,

,

,

,

,

sleep apnea syndromes

,

vestibular diseases

,

,

peripheral nervous system diseases

,

,

,

keratoconjunctivitis sicca

,

dry eye syndromes

,

,

retinal diseases

,

diabetic retinopathy

,

stomach neoplasms

,

pancreatic neoplasms

,

,

,

multiple sclerosis

,

,

amyotrophic lateral sclerosis

,

motor neuron disease

,

,

bipolar disorder

,

post-acute covid-19 syndrome

,

,

,

non-small-cell lung carcinoma

,

precursor cell lymphoblastic leukemia-lymphoma

,

second primary neoplasms

,

type 2 diabetes mellitus

,

chromosome deletion

,

type 1 diabetes mellitus

,

diastolic heart failure

,

,

cystic fibrosis

,

,

renal insufficiency

,

squamous cell carcinoma

,

squamous cell neoplasms

,

,

,

,

burkitt lymphoma

,

,

thrombocytopenia

,

,

lactic acidosis

,

,

mantle-cell lymphoma

,

lymphoid leukemia

,

aids-related lymphoma

,

spinal cord injuries

,

prader-willi syndrome

,

post-cardiac arrest syndrome

,

,

pulmonary tuberculosis

,

rectal neoplasms

,

cri-du-chat syndrome

,

macrocytic anemia

,

,

chronic hepatitis b

,

,

chronic hepatitis

,

,

atopic dermatitis

,

,

,

,

noise-induced hearing loss

,

,

,

sudden death cardiac

,

,

basal cell carcinoma

,

nerve compression syndromes

,

,

charcot-marie-tooth disease

,

hereditary sensory and motor neuropathy

,

,

bipolar and related disorders

,

,

,

metabolic diseases

,

,

glycogen storage disease type ii

,

lysosomal storage diseases

,

glycogen storage disease

,

,

,

,

mitochondrial diseases

,

healthy volunteers/patients

,

parkinson disease

,

colorectal neoplasms

,

chronic fatigue syndrome

,

,

,

breast neoplasms

,

coronary artery disease

,

,

polycystic ovary syndrome

,

prostatic neoplasms

,

cerebellar ataxia

,

huntington disease

,

myocardial ischemia

,

,

,

,

friedreich ataxia

,

,

,

progressive supranuclear palsy

,

duchenne muscular dystrophy

,

muscular dystrophies

,

myelodysplastic syndromes

,

coronary disease

,

drug-related side effects and adverse reactions

,

non-alcoholic fatty liver disease

,

,

cognitive dysfunction

,

cholangiocarcinoma

,

liver neoplasms

,

neoplasm metastasis

,

,

biliary tract neoplasms

,

t-cell lymphoma

,

t-cell lymphoma peripheral

,

,

,

persian gulf syndrome

,

,

hyperlipidemias

,

hyperlipoproteinemias

,

,

,

gastrointestinal neoplasms

,

neuroendocrine tumors

,

carcinoid tumor

,

paraneoplastic syndromes

,

islet cell adenoma

,

malignant carcinoid syndrome

,

rem sleep behavior disorder

,

mental disorders

,

headache disorders

,

nervous system diseases

,

hepatocellular carcinoma

,

gallbladder neoplasms

,

ampulla of vater cancer

,

pathologic dilatation

,

,

corneal diseases

,

,

multiple organ failure

,

,

,

limb-girdle muscular dystrophies

,

,

coronary artery bypass

,

cardiac valve annuloplasty

,

,

coronary stenosis

,

,

juvenile arthritis

,

,

,

,

myotonic dystrophy

,

,

,

cardiovascular diseases

,

male infertility

,

hypercholesterolemia

,

,

migraine disorders

,

diabetes mellitus

,

metabolic syndrome

,

oxidative stress

,

kidney diseases

,

,

atherosclerosis

,

,

,

,

chronic kidney failure

,

insulin resistance

,

,

,

,

hypertriglyceridemia

,

,

chronic renal insufficiency

,

critical illness

,

,

,

opioid-related disorders

,

morphine dependence

,

,

,

,

,

muscle weakness

,

cardiomyopathies

,

dilated cardiomyopathy

,

severe acute respiratory syndrome-related coronavirus

,

severe acute respiratory syndrome

,

,

,

,

temporomandibular joint disorders

,

,

temporomandibular joint dysfunction syndrome

,

acute coronary syndrome

,

attitudes health knowledge practice

,

postoperative pain

,

periodontal diseases

,

,

,

,

,

muscular diseases

,

depressive disorder

,

,

myofascial pain syndromes

,

,

,

medication adherence

,

chromosome disorders

,

autistic disorder

,

autism spectrum disorder

,

,

fetal growth retardation

,

premature birth

,

iron deficiencies

,

vitamin b 12 deficiency

,

,

dermatomyositis

,

,

female infertility

,

,

cardiovascular abnormalities

,

ascorbic acid deficiency

,

ovarian reserve

,

vascular remodeling

,

macular degeneration

,

wet macular degeneration

,

,

post-concussion syndrome

,

gestational diabetes

,

pregnancy outcome

,

gingival recession

,

acute kidney injury

,

intellectual disability

,

hepatic encephalopathy

,

psychotic disorders

,

health status indicators

,

aphthous stomatitis

,

,

,

,

attention deficit disorder with hyperactivity

,

,

mitochondrial genome

,

reproductive physiological phenomena

,

inborn genetic diseases

,

pulmonary arterial hypertension

,

familial primary pulmonary hypertension

,

spontaneous abortion

,

,

kidney transplantation

,

ischemic preconditioning

,

,

respiratory tract infections

,

encephalomyelitis

,

dietary supplements

,

,

,

dental implants

,

,

,

chronic periodontitis

,

,

,

,

physical fitness

,

liver cirrhosis

,

,

st elevation myocardial infarction

,

,

communicable diseases

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

huntington disease

neoplasm metastasis

,

pancreatic neoplasms

,

transitional cell carcinoma

,

,

urologic neoplasms

,

urethral neoplasms

,

ureteral neoplasms

,

pelvic neoplasms

,

stomach neoplasms

,

ovarian neoplasms

,

non-small-cell lung carcinoma

,

prostatic neoplasms

,

invasive hydatidiform mole

,

,

,

urinary bladder neoplasms

,

,

muscle neoplasms

graft vs host disease

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