Drugs in Dev. Genetic Disease
Phase IV
United States Of America 
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
New Biomarker Data Support Potential Benefit of SPINRAZA in Infants
Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
June 03, 2024
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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 ...
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
August 01, 2021
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
July 21, 2020
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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 F508de...
Product Name : Kaftrio
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
July 20, 2020
Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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