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Achieved Primary Endpoint with Sepiapterin in PKU Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC 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Find Clinical Drug Pipeline Developments & Deals by PTC Therapeutics
PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin that acts as
nitric oxide synthase modulator. It is being evaluated for the treatment of Phenylketonuria.
Upstaza (eladocagene exuparvovec) is a gene therapy medicinal product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC). It is being evaluated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
Translarna (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. It is under phase 3 clinical development for the treatment of nonsense mutation Duchenne Muscular Dystrophy.
Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, which is currently being evaluated for the treatment of duchenne muscular dystrophy from a nonsense mutated dystrophin gene, in patients aged 2 or above via oral suspension.
Under the agreement, Royalty Pharma acquires additional royalties on Evrysdi (risdiplam), a survival motor neuron 2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency.
PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Mitochondrial Disease Associated Seizures.
PTC518, is a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression.
Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia.
PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products and has the potential to treat the broad range of PKU patients.
Waylivra (volanesorsen), an antisense oligonucleotide designed to inhibit the formation of apoC-III, is a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology. Waylivra has received conditional marketing approval in the EU as a treatment for FCS.
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