K. Benguerba
8 Papers
K. Benguerba is an academic researcher. The author has contributed to research in topics: Medicine & Spinal muscular atrophy. The author has co-authored 6 publications.
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Papers
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
Laurent Servais,J. W. Day,Darryl C. De Vivo,Janbernd Kirschner,Eugenio Mercuri,Francesco Muntoni,Crystal M. Proud,Perry B. Shieh,Eduardo F. Tizzano,Susana Quijano-Roy,I. Desguerre,Kayoko Saito,Eric Faulkner,K. Benguerba,Dheeraj Raju,Nicole LaMarca,Rui Sun,Fred Anderson,Richard S. Finkel +18 more
TL;DR: These real-world outcomes support findings from the interventional trial program and demonstrate effectiveness of onasemnogene abeparvovec over a large patient population, which was consistent with initial clinical data and published 5-year follow-up data.
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Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.
Crystal M Proud,Eugenio Mercuri,Richard S. Finkel,Janbernd Kirschner,Darryl C. De Vivo,Francesco Muntoni,Kayoko Saito,Eduardo F Tizzano,Isabelle Desguerre,Susana Quijano-Roy,K. Benguerba,Dheeraj Raju,Eric Faulkner,Laurent Servais +13 more
TL;DR: This work validated a rational, systematic approach for defining/grouping survival motor neuron‐targeted disease‐modifying treatment (DMT) scenarios and forms the basis to explore the safety and efficacy profile of the different combinations of DMT in SMA.
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P.103 Onasemnogene abeparvovec (OA) treatment outcomes by patient weight at infusion: Initial findings from the RESTORE registry
L. Servais,K. Benguerba,Darryl C. De Vivo,J. Kirschner,Eduardo de Oliveira Mercuri,F. Muntoni,Crystal M. Proud,E. Tizzano,Susana Quijano-Roy,Kayoko Saito,D Vinay Raju,Nicole M. LaMarca,R. Sun,F. Anderson,E. Faulkner,R. Finkel +15 more
TL;DR: In this article , the authors compared baseline characteristics, effectiveness (measured by CHOP INTEND), and safety of OA for patients weighing <8.5 kg at the time of infusion in the RESTORE SMA patient registry.
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Outcomes in Patients with Spinal Muscular Atrophy and Four or More SMN2 Copies Treated with Onasemnogene Abeparvovec: Findings from RESTORE (P7-9.007)
Richard S. Finkel,K. Benguerba,Manish Gehani,Dheeraj Raju,Eric Faulkner,Nicole M. LaMarca,Laurent Servais +6 more
- 25 Apr 2023
TL;DR: Finkel et al. as mentioned in this paper described clinical outcomes after onasemnogene abeparvovec (OA) monotherapy for patients with ≥4 SMN2> copies in RESTORE, a comprehensive, noninterventional spinal muscular atrophy (SMA) registry.
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P.105 Safety and effectiveness of onasemnogene abeparvovec (OA) alone or with other disease-modifying therapies (DMTs): Findings from RESTORE
L. Servais,K. Benguerba,Darryl C. De Vivo,J. Kirschner,F. Muntoni,Crystal M. Proud,E. Tizzano,Kayoko Saito,D. Raju,Nicole M. LaMarca,R. Sun,F. Anderson,E. Faulkner,R. Finkel +13 more
TL;DR: In this article , the authors describe real-world safety and effectiveness of OA alone or in combination with other DMTs for patients with SMA, and evaluate patients with 2 or 3 SMN2 gene copies.