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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

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

,

,

colorectal neoplasms

,

pancreatic neoplasms

,

,

esophageal neoplasms

,

,

esophageal squamous cell carcinoma

,

biliary tract neoplasms

,

acrocephalosyndactylia

dermatomyositis

,

ulcerative colitis

,

hyperparathyroidism

,

secondary hyperparathyroidism

,

neoplasm metastasis

,

chronic renal insufficiency

,

left ventricular hypertrophy

,

kidney diseases

,

,

,

healthy volunteers/patients

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

Bos taurus adrenal cortex corticosteroids

pharmacokinetics

,

acute-on-chronic liver failure

,

gastrointestinal diseases

,

digestive system diseases

,

aids-related complex

,

primary myelofibrosis

,

myelodysplastic-myeloproliferative diseases

,

pulmonary arterial hypertension

,

chemical and drug induced liver injury

,

respiration disorders

,

myeloproliferative disorders

,

bcr-abl positive chronic myelogenous leukemia

,

leukemia myeloid chronic atypical bcr-abl negative

,

biological availability

,

,

,

respiratory syncytial virus infections

,

,

,

sclerosing cholangitis

,

streptococcus agalactiae

,

,

,

cerebrospinal fluid shunts

,

ventriculoperitoneal shunt

,

retinal diseases

,

,

rheumatoid arthritis

,

,

,

,

brain stem neoplasms

,

overactive urinary bladder

,

chronic hepatitis b

,

essential hypertension

,

,

,

herb-drug interactions

,

,

medical sociology

,

oxidative stress

,

persian gulf syndrome

,

dystonic disorders

,

,

bone marrow transplantation

,

,

,

chinese traditional medicine

,

,

waldenstrom macroglobulinemia

,

paraproteinemias

,

,

phase i metabolic detoxication

,

renal replacement therapy

,

testicular neoplasms

,

neutrophilic leukemia chronic

,

eosinophilic leukemia acute

,

trophoblastic neoplasms

,

short bowel syndrome

,

,

sleep wake disorders

,

,

,

,

,

,

anti-neutrophil cytoplasmic antibody-associated vasculitis

,

autosomal dominant polycystic kidney

,

food-drug interactions

,

,

non-small-cell lung carcinoma

,

neoplasm metastasis

,

brain neoplasms

,

,

,

,

,

triple negative breast neoplasms

,

,

,

male breast neoplasms

,

cocaine-related disorders

,

endometrial neoplasms

,

,

renal cell carcinoma

,

ovarian neoplasms

,

systemic inflammatory response syndrome

,

bipolar disorder

,

,

ovarian epithelial carcinoma

,

synovial sarcoma

,

salivary gland neoplasms

,

cardiovascular diseases

,

rheumatic heart disease

,

castration-resistant prostatic neoplasms

,

bronchopulmonary dysplasia

,

coronary artery disease

,

,

pulmonary sarcoidosis

,

,

multiple sclerosis

,

esophageal neoplasms

,

,

kidney neoplasms

,

uterine cervical neoplasms

,

cerebral ventriculitis

,

pelvic infection

,

,

,

,

,

,

nephrocalcinosis

,

cholangiocarcinoma

,

rhabdomyosarcoma

,

,

,

bile duct neoplasms

,

,

,

,

b-cell lymphoma

,

,

,

respiratory insufficiency

,

periapical abscess

,

central nervous system diseases

,

gastrointestinal neoplasms

,

coronary disease

,

myocardial ischemia

,

chlamydophila infections

,

chlamydial pneumonia

,

randomized controlled trials as topic

,

genomic instability

,

,

male genital diseases

,

,

thyroid neoplasms

,

,

uterine neoplasms

,

macular degeneration

,

subacute bacterial endocarditis

,

hemangiosarcoma

,

pathologic complete response

,

pelvic inflammatory disease

,

psychotic disorders

,

genetic skin diseases

,

myeloid leukemia

,

inborn genetic diseases

,

left ventricular dysfunction

,

graft vs host disease

,

urologic neoplasms

,

neurofibrosarcoma

,

pulmonary blastoma

,

urologic diseases

,

fallopian tube neoplasms

,

systemic scleroderma

,

intestinal pseudo-obstruction

,

cerebral intraventricular hemorrhage

,

periventricular leukomalacia

,

,

maxillofacial injuries

,

appendiceal neoplasms

,

pseudomyxoma peritonei

,

,

,

primitive neuroectodermal tumors

,

primitive neuroectodermal tumors peripheral

,

alveolar rhabdomyosarcoma

,

embryonal rhabdomyosarcoma

,

neuroectodermal tumors

,

endometrioid carcinoma

,

,

squamous cell carcinoma of head and neck

,

squamous cell carcinoma

,

oropharyngeal neoplasms

,

unknown primary neoplasms

,

squamous cell neoplasms

,

tongue neoplasms

,

verrucous carcinoma

,

paranasal sinus neoplasms

,

laryngeal diseases

,

gallbladder neoplasms

,

urethral neoplasms

,

transitional cell carcinoma

,

ureteral neoplasms

,

,

finger injuries

,

tobacco use disorder

,

,

,

tuberculous pericarditis

,

,

gardnerella vaginalis

,

central serous chorioretinopathy

,

genetic predisposition to disease

,

tibial meniscus injuries

,

cartilage fractures

,

,

ischemic stroke

,

,

cerebrovascular disorders

,

pancreatic fistula

,

pregnancy in obesity

,

,

cephalosporin resistance

,

,

congenital syphilis

,

,

,

multiple myeloma

,

parkinson disease

,

aids-related opportunistic infections

,

bacterial skin diseases

,

meningeal tuberculosis

,

gram-positive bacterial infections

,

colorectal neoplasms

,

,

myelodysplastic syndromes

,

kidney diseases

,

respiratory tract diseases

,

hodgkin disease

,

inflammatory bowel diseases

,

chlamydia infections

,

bacterial vaginosis

,

large-cell lymphoma anaplastic

,

multidrug-resistant tuberculosis

,

large b-cell lymphoma diffuse

,

plasma cell neoplasms

,

b-cell lymphoma marginal zone

,

b-cell chronic lymphocytic leukemia

,

treponemal infections

,

erectile dysfunction

,

invasive hydatidiform mole

,

follicular lymphoma

,

hematopoietic stem cell transplantation

,

alcoholic hepatitis

,

myelomonocytic leukemia chronic

,

,

,

head and neck neoplasms

,

post-lyme disease syndrome

,

periodontal pocket

,

hidradenitis suppurativa

,

alzheimer disease

,

sickle cell anemia

,

,

,

,

,

,

hematologic diseases

,

mantle-cell lymphoma

,

burkitt lymphoma

,

asymptomatic diseases

,

,

,

,

myeloid leukemia chronic-phase

,

myeloid leukemia accelerated phase

,

,

,

reactive arthritis

,

,

neurodevelopmental disorders

,

microscopic colitis

,

microbial drug resistance

,

systolic heart failure

,

iatrogenic disease

,

,

gastrointestinal motility

,

,

esophageal achalasia

,

sezary syndrome

,

prolymphocytic leukemia

,

mycosis fungoides

,

t-cell lymphoma cutaneous

,

large-cell lymphoma immunoblastic

,

intestinal perforation

,

,

intestinal obstruction

,

,

bacterial infections and mycoses

,

laryngeal neoplasms

,

,

treatment outcome

,

,

,

bacillary dysentery

,

postoperative pain

,

nervous system diseases

,

pancreaticoduodenectomy

,

sexually transmitted diseases

,

human influenza

,

falciparum malaria

,

,

uterine cervicitis

,

,

collagenous colitis

,

,

environmental biomarkers

,

amyotrophic lateral sclerosis

,

motor neuron disease

,

nasal obstruction

,

nasal surgical procedures

,

sick sinus syndrome

,

,

,

hemopneumothorax

,

,

pancreatic diseases

,

autonomic nervous system diseases

,

,

biphenotypic leukemia acute

,

immunoblastic lymphadenopathy

,

hairy cell leukemia

,

intraocular lymphoma

,

precursor t-cell lymphoblastic leukemia-lymphoma

,

large granular lymphocytic leukemia

,

hepatorenal syndrome

,

alcoholic liver diseases

,

congenital abnormalities

,

vesico-ureteral reflux

,

peptic ulcer perforation

,

peptic ulcer hemorrhage

,

endoscopic mucosal resection

,

,

mechanical ventilators

,

fecal microbiota transplantation

,

multiple system atrophy

,

,

,

,

induced hypothermia

,

suppurative otitis media

,

cholecystectomy

,

central nervous system neoplasms

,

intestinal neoplasms

,

nervous system neoplasms

,

bites and stings

,

intestinal diseases

,

pseudomembranous enterocolitis

,

hemoglobinopathies

,

agammaglobulinemia

,

inflammatory breast neoplasms

,

patient preference

,

incisional hernia

,

,

,

mycobacterium tuberculosis

,

lymph node tuberculosis

,

adolescent development

,

artificial respiration

,

intellectual disability

,

,

subdural hematoma chronic

,

liver transplantation

,

,

healthy volunteers/patients

,

communicable diseases

,

,

breast neoplasms

,

,

urinary tract infections

,

bacterial infections

,

,

helicobacter infections

,

,

surgical wound infection

,

,

pulmonary tuberculosis

,

,

ventilator-associated pneumonia

,

intraabdominal infections

,

liver cirrhosis

,

,

wound infection

,

,

cystic fibrosis

,

bacterial pneumonia

,

hepatic encephalopathy

,

,

,

,

respiratory tract infections

,

,

,

staphylococcal infections

,

,

drug interactions

,

,

critical illness

,

,

community-acquired pneumonia

,

,

antibiotic prophylaxis

,

,

febrile neutropenia

,

acquired immunodeficiency syndrome

,

irritable bowel syndrome

,

,

mycobacterium avium-intracellulare infection

,

,

,

hypersensitivity

,

,

,

,

,

,

,

infectious skin diseases

,

,

healthcare-associated pneumonia

,

postoperative complications

,

premature birth

,

,

,

hematologic neoplasms

,

non-hodgkin lymphoma

,

pseudomonas infections

,

gram-negative bacterial infections

,

,

latent tuberculosis

,

soft tissue infections

,

,

,

renal insufficiency

,

,

,

premature rupture fetal membranes

,

mycobacterium infections

,

severe acute malnutrition

,

stomach neoplasms

,

diabetes mellitus

,

,

,

prosthesis-related infections

,

wounds and injuries

,

neonatal sepsis

,

prostatic neoplasms

,

,

,

escherichia coli infections

,

drug hypersensitivity

,

methicillin-resistant staphylococcus aureus

,

,

acute kidney injury

,

disease susceptibility

,

enterobacteriaceae infections

,

,

urinary bladder neoplasms

,

,

infectious arthritis

,

,

,

cross infection

,

chronic renal insufficiency

,

,

,

chronic periodontitis

,

,

,

nontuberculous mycobacterium infections

,

,

,

,

,

,

cesarean section

,

myeloid leukemia acute

,

pancreatic neoplasms

,

,

,

staphylococcal skin infections

,

mycobacterium avium complex

,

chronic obstructive pulmonary disease

,

chronic bronchitis

,

,

klebsiella infections

,

,

esophageal and gastric varices

,

,

type 2 diabetes mellitus

,

aggressive periodontitis

,

,

periodontal diseases

,

,

chronic kidney failure

,

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

,

cardiac arrhythmias

,

,

community-acquired infections

,

streptococcal infections

,

rectal neoplasms

,

central nervous system infections

,

ulcerative colitis

,

,

,

lyme neuroborreliosis

,

abdominal abscess

,

,

,

staphylococcus aureus

,

,

,

drug-related side effects and adverse reactions

,

,

,

,

,

endophthalmitis

,

chorioamnionitis

,

necrotizing enterocolitis

,

hepatic insufficiency

,

,

pseudomonas aeruginosa

,

obstructive lung diseases

,

insulin resistance

,

non-alcoholic fatty liver disease

,

hepatocellular carcinoma

,

,

,

,

bacterial drug resistance

,

nephrolithiasis

,

,

autoimmune diseases

,

,

respiratory aspiration

,

diverticular diseases

,

,

portal hypertension

,

,

,

,

hemostatic disorders

,

gastrointestinal microbiome

,

premature obstetric labor

,

,

liver neoplasms

,

,

bacterial endocarditis

,

benign migratory glossitis

,

erythema chronicum migrans

,

,

gastrointestinal hemorrhage

,

mental disorders

,

acalculous cholecystitis

,

,

pregnancy complications

,

surgical amputation

,

uterine cervical incompetence

,

kidney transplantation

,

,

acute cholecystitis

,

bacterial meningitis

,

,

dental implants

,

therapeutic equivalency

,

bariatric surgery

,

peri-implantitis

,

,

inguinal hernia

,

tooth extraction

,

child nutrition disorders

,

obstetric labor complications

,

opioid-related disorders

,

,

coagulation protein disorders

,

puerperal infection

,

obsessive-compulsive disorder

,

colonic neoplasms

,

pyogenic liver abscess

,

pneumococcal pneumonia

,

,

mandibular fractures

,

humeral fractures

,

,

dental focal infection

,

focal infection

,

acinetobacter infections

,

osteoarticular tuberculosis

,

infectious pregnancy complications

,

atopic dermatitis

,

,

,

prostatic hyperplasia

,

immediate hypersensitivity

,

continuous ambulatory peritoneal dialysis

,

colonic diverticulitis

,

implant capsular contracture

,

peritoneal dialysis

,

,

prophylactic surgical procedures

,

paratuberculosis

,

,

acute necrotizing pancreatitis

,

thrombocytopenic purpura idiopathic

,

thrombocytopenic purpura

,

,

,

,

out-of-hospital cardiac arrest

,

,

aspiration pneumonia

,

,

,

disease progression

,

,

clostridium infections

,

gastroenteritis

,

,

spontaneous rupture

,

child development

,

coronary thrombosis

,

,

adaptive immunity

,

replacement arthroplasty knee

,

postpartum hemorrhage

,

pituitary neoplasms

,

pleural effusion

,

,

breast implantation

,

,

,

colonic diverticulosis

,

knee osteoarthritis

,

glucose metabolism disorders

,

glucose intolerance

,

,

obstructive sleep apnea

,

sleep apnea syndromes

,

,

,

,

biliary liver cirrhosis

,

patient compliance

,

antibody specificity

,

,

pathologic processes

,

thrombocytopenia

,

gastroesophageal reflux

,

perinatal death

,

carpal tunnel syndrome

,

vitreoretinal surgery

,

aortic valve disease

,

congenital heart defects

,

,

,

cutaneous tuberculosis

,

common variable immunodeficiency

,

acute coronary syndrome

,

,

spinal tuberculosis

,

,

,

,

otitis media with effusion

,

compulsive personality disorder

,

neisseria gonorrhoeae

,

,

surgical wound dehiscence

,

,

,

,

,

pulmonary infarction

,

haemophilus infections

,

,

high-risk pregnancy

,

borrelia infections

,

,

blood platelet disorders

,

ankylosing spondylitis

,

spondylarthritis

,

,

urinary diversion

,

catheter-related infections

,

multiple drug resistance

,

hereditary congenital and neonatal diseases and abnormalities

,

,

urinary incontinence

,

stress urinary incontinence

,

mycobacterium abscessus

,

internal fracture fixation

,

drug monitoring

,

,

sleep initiation and maintenance disorders

,

,

peritonsillar abscess

,

end stage liver disease

,

,

,

,

,

hyperaldosteronism

,

,

,

neurotoxicity syndromes

,

penicillin resistance

,

,

,

,

protein-energy malnutrition

,

,

closed fractures

,

,

,

periapical periodontitis

,

gingival recession

,

replacement arthroplasty hip

,

,

cognitive dysfunction

,

coronavirus infections

,

,

mucocutaneous lymph node syndrome

,

hyperphosphatemia

,

adrenal insufficiency

,

attention deficit disorder with hyperactivity

,

cytokine release syndrome

,

viral pneumonia

,

,

,

mycoplasma pneumonia

,

mycoplasma infections

,

pleuropneumonia

,

,

propionibacterium acnes

,

aseptic meningitis

,

depressive disorder

,

salmonella infections

,

hiv seropositivity

,

,

spontaneous abortion

,

incomplete abortion

,

bisphosphonate-associated osteonecrosis of the jaw

,

retained placenta

,

dentofacial deformities

,

cerebral hemorrhage

,

subarachnoid hemorrhage

,

hemodiafiltration

,

,

epidemic louse-borne typhus

,

,

,

thyroid diseases

,

parathyroid diseases

,

continuous renal replacement therapy

,

migraine disorders

,

,

,

,

,

,

general anesthesia

,

,

,

neuromuscular blockade

,

,

,

cytomegalovirus retinitis

,

adenoviridae infections

,

port-wine stain

,

,

,

capillary hemangioma

,

autistic disorder

,

,

treatment-resistant schizophrenia

,

potassium deficiency

,

stem cell transplantation

,

asymptomatic infections

,

respiratory distress syndrome

,

intestinal fistula

,

,

precancerous conditions

,

metabolic syndrome

,

retinal detachment

,

dissociative disorders

,

,

choledocholithiasis

,

,

velopharyngeal insufficiency

,

,

,

,

,

elective surgical procedures

,

hirschsprung disease

,

,

,

wasting syndrome

,

body weight changes

,

,

stomach diseases

,

,

,

uterine prolapse

,

,

,

,

glomerulonephritis

,

membranous glomerulonephritis

,

nephrotic syndrome

,

,

venous thrombosis

,

,

,

dumping syndrome

,

,

implantable defibrillators

,

hepatopulmonary syndrome

,

,

treatment failure

,

,

listeria monocytogenes

,

,

segmental mastectomy

,

thoracic surgical procedures

,

chronic hepatitis c

,

multiple organ failure

,

,

delayed hypersensitivity

,

vaginal diseases

,

,

disorders of excessive somnolence

,

hypercholesterolemia

,

,

,

newborn respiratory distress syndrome

,

,

corneal diseases

,

,

,

accidental injuries

,

recreational drug use

,

laboratory infection

,

vascular access devices

,

pneumococcal infections

,

,

biliary atresia

,

,

hip osteoarthritis

,

pelvic organ prolapse

,

laparoscopic cholecystectomy

,

cardiogenic shock

,

,

residual neoplasm

,

acinetobacter baumannii

,

conservative treatment

,

blood coagulation disorders

,

,

maxillary sinusitis

,

pregnancy outcome

,

,

,

,

alveolar ridge augmentation

,

,

heart valve diseases

,

rheumatic fever

,

,

,

emergence delirium

,

ovarian neoplasms

,

non-small-cell lung carcinoma

,

endometrial neoplasms

,

stomach neoplasms

,

head and neck neoplasms

,

esophageal neoplasms

,

,

neoplasm metastasis

1

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