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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
ataxia
,
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
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
parkinson disease
,
multiple system atrophy
,
lewy body disease
,
dementia
,
synucleinopathies
,
progressive supranuclear palsy
,
corticobasal degeneration
Alfacalcidol
hemophilia a
,
blood coagulation disorders
,
hemostatic disorders
,
type 2 diabetes mellitus
,
diabetes mellitus
Aspartame
healthy volunteers/patients
,
colitis
,
ulcer
,
obesity
,
subarachnoid hemorrhage
,
crohn disease
,
von willebrand diseases
,
ulcerative colitis
,
hodgkin disease
,
constipation
,
cytomegalovirus infections
,
cytomegalovirus
,
hemophilia a
,
precocious puberty
,
type 2 diabetes mellitus
,
schizophrenia
Bevifimod
allergic rhinitis seasonal
,
nasal obstruction
,
sneezing
,
dental anxiety
,
restless legs syndrome
,
spinal cord injuries
,
type 1 diabetes mellitus
,
hiv infections
,
non-small-cell lung carcinoma
,
osteoarthritis
,
breast neoplasms
,
depression
,
neurologic manifestations
,
wounds and injuries
,
central nervous system diseases
,
spinal cord diseases
,
sleep wake disorders
,
inguinal hernia
,
peripheral nervous system diseases
,
radiculopathy
,
psoriasis
,
fibromyalgia
,
healthy volunteers/patients
,
neuralgia
,
partial epilepsies
,
diabetic neuropathies
,
epilepsy
,
pain
,
sleep initiation and maintenance disorders
,
seizures
,
postherpetic neuralgia
,
postoperative pain
,
anxiety disorders
,
low back pain
,
generalized anxiety disorder
,
complex partial epilepsy
,
back pain
,
breast feeding
,
bronchiolitis obliterans syndrome
,
neoplasms
,
bone neoplasms
,
intractable pain
,
phantom limb
,
burns
,
renal insufficiency
,
pharmacovigilance
Unesbulin
ovarian neoplasms
,
ovarian epithelial carcinoma
,
glioma
,
diffuse intrinsic pontine glioma
,
neoplasms
,
leiomyosarcoma
Bos taurus adrenal cortex corticosteroids
graft vs host disease
,
bronchiolitis obliterans syndrome
Lactams
neoplasms
,
chronic renal insufficiency
,
kidney diseases
,
type 2 diabetes mellitus
,
rheumatoid arthritis
,
healthy volunteers/patients
,
constipation
,
non-small-cell lung carcinoma
,
breast neoplasms
,
sepsis
,
septic shock
,
infections
,
pneumonia
,
bacterial pneumonia
Mk3475
pancreatic ductal carcinoma
,
mycoses
,
squamous cell carcinoma
,
merkel cell carcinoma
,
mycosis fungoides
,
squamous cell neoplasms
,
neoplasms
,
colorectal neoplasms
,
colonic neoplasms
,
rectal neoplasms
,
sarcoma
,
breast neoplasms
,
alveolar soft part sarcoma
,
brain neoplasms
,
triple negative breast neoplasms
,
hodgkin disease
,
skin neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
renal cell carcinoma
,
carcinoma
,
melanoma
,
neoplasm metastasis
Solumedrol
atrial fibrillation
,
chronic renal insufficiency
,
chronic kidney failure
,
kidney transplantation
,
pulmonary fibrosis
,
secondary hyperparathyroidism
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