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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:
The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508del mutation.
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