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PTC 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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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
Ataluren
healthy volunteers/patients
,
aniridia
,
hemophilia a
,
hemophilia b
,
colorectal neoplasms
,
endometrial neoplasms
,
epilepsy
,
myoclonic epilepsies
,
inborn errors metabolism
,
inborn errors amino acid metabolism
,
duchenne muscular dystrophy
,
muscular dystrophies
,
fibrosis
,
cystic fibrosis
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
neuromuscular diseases
,
atrophic muscular disorders
Deflazacort
healthy volunteers/patients
,
renal insufficiency
,
liver diseases
,
hepatic insufficiency
,
muscular dystrophies
,
duchenne muscular dystrophy
,
proteinuria
,
membranous glomerulonephritis
,
aspergillosis
,
allergic bronchopulmonary aspergillosis
,
pulmonary aspergillosis
,
limb-girdle muscular dystrophies
,
inborn genetic diseases
,
muscular diseases
,
nervous system diseases
,
musculoskeletal diseases
,
x-linked genetic diseases
,
neuromuscular diseases
,
atrophic muscular disorders
,
chronic renal insufficiency
,
postoperative pain
,
trismus
,
impacted tooth
,
kidney diseases
,
iga glomerulonephritis
,
immunosuppression therapy
,
asthma
ELADOCAGENE EXUPARVOVEC
argininosuccinic aciduria
Sepiapterin
gastroparesis
,
phenylketonurias
Vatiquinone
tic disorders
,
tourette syndrome
,
tics
,
mitochondrial diseases
,
rett syndrome
,
muscular diseases
,
syndrome
,
inborn errors lipid metabolism
,
noise-induced hearing loss
,
angelman syndrome
,
autistic disorder
,
autism spectrum disorder
,
ataxia
,
friedreich ataxia
,
cerebellar ataxia
,
parkinson disease
,
optic nerve diseases
,
emergencies
,
hereditary optic atrophy leber
Emvododstat
neoplasms
,
breast neoplasms
,
central nervous system neoplasms
,
nervous system neoplasms
,
leukemia
,
myeloid leukemia acute
,
myeloid leukemia
,
sarcoma
,
kaposi sarcoma
,
neurofibromatosis 2
,
neurofibromatosis 1
,
neurofibromatoses
,
neurofibroma
,
covid-19
,
coronavirus infections
,
coronavirus
,
pneumonia
Gilvetmab
prostatic neoplasms
,
small cell carcinoma
,
neuroendocrine tumors
Alfacalcidol
polycythemia vera
,
polycythemia
,
leukemia
,
bcr-abl positive chronic myelogenous leukemia
,
myeloid leukemia
Aspartame
healthy volunteers/patients
,
renal insufficiency
,
chronic kidney failure
,
myeloid leukemia acute
,
immunoassay
,
myelodysplastic syndromes
,
non-hodgkin lymphoma
,
hiv
,
acquired immunodeficiency syndrome
,
immunologic deficiency syndromes
,
acute disease
,
virus diseases
,
respiratory syncytial virus infections
,
leukemia
,
precursor cell lymphoblastic leukemia-lymphoma
,
respiratory syncytial viruses
,
venous thrombosis
,
precursor t-cell lymphoblastic leukemia-lymphoma
,
lymphoid leukemia
,
smoldering multiple myeloma
,
hiv-1
,
neoplasm metastasis
,
chronic hepatitis c
,
breast neoplasms
,
postmenopause
,
peripheral nervous system diseases
,
hematologic neoplasms
,
colorectal neoplasms
,
myositis
,
graft vs host disease
,
bronchiolitis obliterans syndrome
,
multiple myeloma
,
plasma cell neoplasms
,
psoriasis
,
non-small-cell lung carcinoma
,
hiv infections
,
neoplasms
,
infections
,
communicable diseases
,
heart failure
,
covid-19
,
stroke
,
lymphoma
,
ovarian neoplasms
,
juvenile arthritis
,
thromboembolism
,
respiratory tract infections
,
depression
,
major depressive disorder
,
depressive disorder
,
treatment-resistant depressive disorder
,
fallopian tube neoplasms
,
peritoneal neoplasms
,
ovarian epithelial carcinoma
,
inflammatory bowel diseases
,
b-cell chronic lymphocytic leukemia
,
recurrence
,
respiratory insufficiency
,
rheumatic diseases
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
tinea pedis
,
psychotic disorders
,
bipolar disorder
,
psychotic affective disorders
,
mood disorders
,
mental disorders
,
human influenza
,
venous thromboembolism
,
kidney transplantation
,
schizophrenia
,
type 2 diabetes mellitus
,
crohn disease
,
prostatic neoplasms
,
urogenital neoplasms
,
male genital diseases
,
male genital neoplasms
,
genital diseases
,
lung neoplasms
,
hyperkinesis
,
attention deficit disorder with hyperactivity
,
cognitive dysfunction
,
thyroid neoplasms
,
papillary thyroid cancer
,
thyroid diseases
,
goiter
,
obesity
,
retinal diseases
,
ankle injuries
,
ankle fractures
,
fibula fractures
Bevifimod
prostatic neoplasms
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
macular edema
,
edema
,
uveitis
Lactams
renal insufficiency
,
breast neoplasms
,
overactive urinary bladder
,
soft tissue infections
,
pelvic infection
,
urologic diseases
,
hematologic neoplasms
,
neoplasms
,
urinary tract infections
,
pyelonephritis
,
surgical wound infection
,
bacterial pneumonia
,
bacterial infections
,
diabetic foot
,
bacteremia
,
infections
,
communicable diseases
,
intraabdominal infections
,
sepsis
,
pneumonia
,
healthy volunteers/patients
,
febrile neutropenia
,
tuberculosis
,
endometritis
,
puerperal infection
,
prostatic hyperplasia
,
hypersensitivity
,
immediate hypersensitivity
,
chronic kidney failure
,
continuous ambulatory peritoneal dialysis
,
neutropenia
,
fever
,
hyperthermia
,
ventilator-associated pneumonia
,
contraception
,
critical illness
,
obesity
Mk3475
melanoma
Solumedrol
ovarian neoplasms
,
arteritis
,
takayasu arteritis
,
aortic arch syndromes
,
giant cell arteritis
,
ulcerative colitis
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