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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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Quarterly
Annual
Annual
Projections
*Quarterly sales USD (in millions)
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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
,
limb-girdle muscular dystrophies
ELADOCAGENE EXUPARVOVEC
argininosuccinic aciduria
Sepiapterin
gastroparesis
,
phenylketonurias
Vatiquinone
tic disorders
,
tourette syndrome
,
tics
,
alzheimer disease
,
dementia
,
muscular diseases
,
rett syndrome
,
ataxia
,
inborn genetic diseases
,
retinal diseases
,
homocystinuria
,
syndrome
,
inborn errors lipid metabolism
,
noise-induced hearing loss
,
angelman syndrome
,
autistic disorder
,
autism spectrum disorder
,
cerebellar ataxia
,
parkinson disease
,
mitochondrial diseases
,
friedreich ataxia
,
leigh disease
,
melas syndrome
,
diffuse cerebral sclerosis of schilder
,
drug resistant epilepsy
,
merrf syndrome
,
seizures
,
status epilepticus
,
nerve degeneration
,
mitochondrial encephalomyopathies
,
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
urologic neoplasms
,
urethral neoplasms
,
transitional cell carcinoma
,
ureteral neoplasms
,
pelvic neoplasms
,
prostatic neoplasms
,
urinary bladder neoplasms
Alfacalcidol
carcinoma
,
non-small-cell lung carcinoma
,
lung neoplasms
,
small cell carcinoma
,
squamous cell carcinoma
,
squamous cell neoplasms
,
adenocarcinoma of lung
,
large cell carcinoma
Aspartame
healthy volunteers/patients
,
laron syndrome
,
pituitary dwarfism
,
leukemia
,
myelodysplastic syndromes
,
myeloid leukemia acute
,
megakaryoblastic leukemia acute
,
monocytic leukemia acute
,
myelomonocytic leukemia acute
,
myeloid leukemia
,
erythroblastic leukemia acute
,
neoplasms
,
dyspnea
,
asthma
,
chronic obstructive pulmonary disease
,
lung diseases
,
obstructive lung diseases
,
inflammation
Bevifimod
b-cell lymphoma marginal zone
,
follicular lymphoma
,
mantle-cell lymphoma
,
waldenstrom macroglobulinemia
,
interdigitating dendritic cell sarcoma
,
b-cell chronic lymphocytic leukemia
,
leukemia
,
lymphoma
,
lymphoid leukemia
,
non-hodgkin lymphoma
,
b-cell lymphoma
,
non-small-cell lung carcinoma
,
hematologic diseases
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
keratoconjunctivitis sicca
,
dry eye syndromes
,
eye diseases
Lactams
hiv infections
,
tuberculosis
,
acquired immunodeficiency syndrome
,
aids-related opportunistic infections
,
aids-related complex
Mk3475
malignant mesothelioma
,
renal cell carcinoma
,
breast neoplasms
,
uterine cervical neoplasms
,
neoplasms
,
melanoma
,
lung neoplasms
,
carcinoma
,
head and neck neoplasms
,
respiratory tract diseases
,
germ cell and embryonal neoplasms
,
small cell lung carcinoma
,
lung diseases
,
respiratory tract neoplasms
,
bronchogenic carcinoma
,
thoracic neoplasms
,
bronchial diseases
,
neoplasms by histologic type
,
neoplasms by site
,
nerve tissue neoplasms
,
non-small-cell lung carcinoma
,
adenocarcinoma
,
adenocarcinoma of lung
Solumedrol
lymphoma
,
large b-cell lymphoma diffuse
,
b-cell lymphoma
,
leukemia
,
b-cell chronic lymphocytic leukemia
,
recurrence
,
graft vs host disease
,
bronchiolitis obliterans syndrome
,
prostatic neoplasms
,
castration-resistant prostatic neoplasms
,
x-linked bulbo-spinal atrophy
,
follicular lymphoma
,
pulmonary sarcoidosis
1
2
>
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