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            Lead Product(s): Nusinersen

            Therapeutic Area: Genetic Disease Product Name: Spinraza

            Highest Development Status: Phase IV Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2021

            Details:

            The RESPOND study aims to evaluate if Spinraza can benefit SMA patients who have experienced a ‘suboptimal’ response to Zolgensma (onasemnogene abeparvovec). It will be conducted at around 20 sites globally, with researchers aiming to enrol up to 60 children with SMA.

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            Lead Product(s): Nusinersen

            Therapeutic Area: Genetic Disease Product Name: Spinraza

            Highest Development Status: Phase IV Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 21, 2020

            Details:

            The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its continuous production of SMN protein may also benefit patients previously treated with gene therapy.

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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Phase IV Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

            Details: