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    [{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ataluren","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules for Oral Suspension","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ataluren","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"PTC 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                            01

                            PTC Therapeutics

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                            PTC Therapeutics

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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            CHMP Maintains Negative Opinion on Translarna™ Reexamination

                            Details : Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, indicated for the treatment of nonsense mutated duchenne muscular dystrophy , in patients aged 2 or above.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 18, 2024

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            PTC Therapeutics

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                            Fi Europe 2024
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                            PTC Therapeutics

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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            CHMP Issues Negative Opinion On Translarna™ Renewal

                            Details : Translarna (ataluren) is a protein restoration therapy under phase 3 development for nonsense mutation Duchenne Muscular Dystrophy, enabling functioning protein formation in patients.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            January 25, 2024

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            PTC Therapeutics

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                            PTC Therapeutics

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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PTC Therapeutics Provides Updates on Translarna Regulatory Activities

                            Details : 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 s...

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            December 05, 2023

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed

                            PTC Therapeutics

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                            Fi Europe 2024
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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Blackstone Life Sciences

                            Deal Size : $50.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            PTC Therapeutics Announces Strategic Financing Collaboration with Blackstone with up to $1 Billion in Funding

                            Details : The strategic financing will support the acceleration of PTC’s robust and diversified pipeline having lead asset Translarna (ataluren), business development opportunities and general corporate purposes.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            October 27, 2022

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Blackstone Life Sciences

                            Deal Size : $50.0 million

                            Deal Type : Collaboration

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                            05

                            PTC Therapeutics

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                            Fi Europe 2024
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                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PTC Therapeutics to Present Clinical and Real-World Evidence on Translarna™ at the Annual Congress of the Wo...

                            Details : Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 11, 2022

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            PTC Therapeutics

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                            PTC Therapeutics

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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : NICE

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            NICE Review Does Not Recommend Routine Funding For Duchenne Muscular Dystrophy Gene Therapy Ataluren In Draft ...

                            Details : Translarna (ataluren) is the first licensed treatment for DMD that addresses the loss of dystrophin. It works by allowing the body to read over the mutation in the DNA and continue to produce dystrophin.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            September 30, 2022

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : NICE

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna™ (ataluren) in Nonsens...

                            Details : Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 20, 2022

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            PTC Therapeutics

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                            Fi Europe 2024
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                            PTC Therapeutics

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                            Fi Europe 2024
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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PTC Announces Expansion of Translarna™ Label to Include Ambulatory Patients as Young as 2 Years Old in Brazi...

                            Details : Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna's potential to slow disease progression and improve patient outcomes.

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 25, 2021

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutatio...

                            Details : Real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of ca...

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            September 20, 2021

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed

                            PTC Therapeutics

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                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PTC Therapeutics to Host Call to Review Results from its Study of Translarna in Nonsense Mutation Duchenne Mus...

                            Details : PTC Therapeutics is planning to hold talks with the US FDA on approval after the latest Phase II Study 045 of its drug, Translarna (ataluren), failed to significantly improve dystrophin expression in patients with nonsense mutation Duchenne muscular dyst...

                            Brand Name : Translarna

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            February 04, 2021

                            Lead Product(s) : Ataluren

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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