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• W3163227356 abstract "Objective: To integrate and significantly expand available real-world data on patients with spinal muscular atrophy (SMA) by establishing RESTORE – a comprehensive registry of patients with SMA specifically designed to overcome the recognized limitations of existing single-product registries (eg, dissimilarities in data collection, lack of cross-registry patient continuity, and limited access by researchers). Background: SMA is a rapidly progressive, debilitating disease characterized by motor neuron loss, muscle weakness, respiratory failure, and early death. While recent advancements in SMA treatment have dramatically improved prognosis, real-world data on treatment outcomes remain limited – particularly for patients who switch treatments. Design/Methods: The RESTORE Registry is a prospective, multicenter, multinational, observational study that will assess outcomes (eg, survival, pulmonary, nutritional, and motor milestones, functional status, quality of life, adverse events) in patients with all types of SMA (including presymptomatic patients), inform patients, caregivers, regulatory agencies, and researchers on the effectiveness and safety of approved and emerging treatments, and collect information on healthcare resource utilization and caregiver burden. Participating centers may be involved in existing registries or recruited de novo. Data from patients enrolled in partnering registries (eg, iSMAC, TreatNMD, NeuroNEXT, Cure-SMA, SMArtCARE) and the onasemnogene abeparvovec-xioi (formerly AVXS-101) managed access program are transferred to the RESTORE database. Follow-up duration is 15 years from enrollment or until death. Results: As of 14 October 2019, 52 patients – including some who received onasemnogene abeparvovec subsequent to other available therapies – have been enrolled across 19 active sites in the United States. The RESTORE Registry is rapidly expanding globally, with 48 sites in start-up. Initial data are anticipated in Q4 2019. Conclusions: RESTORE, a comprehensive, treatment-agnostic SMA patient registry, will allow extended assessments of patient outcomes and SMA interventions. Initial data will include information on patients receiving onasemnogene abeparvovec subsequent to other available therapies. Disclosure: Dr. Servais has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Avexis, Inc., Biogen, Biophytis, Cytokinetics, Dynacure, Roche, Santhera, Sarepta Therapeutics. Dr. Servais has received research support from Avexis, Inc., Biogen, Dynacure, and Roche. Dr. Day has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Consultant for AMO Pharma, AveXis, Biogen, Cytokinetics, Ionis Pharmaceuticals, Inc., Pfizer, Roche, Santhera, Sarepta.. Dr. Day has received royalty, license fees, or contractual rights payments from Patents licensed to Athena Diagnostics for genetic testing of myotonic dystrophy type 2 (US patent 7442782) and spinocerebellar ataxia type 5 (US patent 7527931). Dr. Day has received research support from Grants from AMO Pharma, aTyr, AveXis, Biogen, Bristol-Myers Squibb, Cytokinetics, Ionis Pharmaceuticals, Inc., Roche, Sanofi-Genzyme, Sarepta.. Dr. De Vivo has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisor/consultant for AveXis, Biogen, Cytokinetics, Ionis Pharmaceuticals, Metafora, Roche, Sanofi-Aventis, Santhera, Sarepta, Scholar Rock, PTC, Ultragenyx, Cure SMA and the SMA Foundation. Dr. De Vivo has received research support from Grants/clinical trial support from Biogen, CureSMA, Hope for Children Research Foundation, Mallinckrodt, the National Institutes of Health, PTC, Santhera, Sarepta, Scholar Rock, the SMA Foundation, Ultragenyx, NINDS, Glut1 Deficiency Foundation, and the U. Dr. Kirschner has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for SMA studies for AveXis, Biogen, Ionis Pharmaceuticals, Inc., Novartis, and Roche.. Dr. Kirschner has received research support from Research grant from Avexis, Biogen, PTC, Roche, Santhera, and Sarepta.. Dr. Mercuri has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for SMA studies for AveXis, Biogen, Ionis Pharmaceuticals, Inc., Novartis, and Roche.. Dr. Mercuri has received research support from Principal Investigator for ongoing Biogen/Ionis Pharmaceuticals, Inc. and Roche clinical trials; support from Biogen for a natural history registry; funding from Famiglie SMA Italy, Italian Telethon, and SMA Europe.. Dr. Muntoni has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for AveXis, Biogen, Cytokinetics, Novartis, Pfizer, PTC, Roche, Sarepta, Summit, and Wave. Dr. Muntoni has received research support from Principal Investigator for ongoing Ionis Pharmaceuticals, Inc./Biogen and Roche clinical trials; funding from Muscular Dystrophy UK, SMA Europe, and SMA Trust UK. Dr. Shieh has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes, Sarepta, Pfizer, Genentech, Avexis, Biogen, Catalyst, Argenyx, Alexion, CSL Behring, Grifols. Dr. Shieh has received research support from Sarepta, Pfizer, Audentes, Avexis, Biogen, PTC, Roche, Sanofi, Reveragen, Acceleron.Dr. Tizzano has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Consultant for AveXis, Biogen, Biologix, Cytokinetics, and Roche; scientific/medical advisor to FAME Chile, Familias SMA Argentina, FUNDAME, SMA Europe, and TREAT-NMD. Dr. Tizzano has received research support from Grant support from Biogen and Ionis Pharmaceuticals, Inc.; funding from Fundacion Privada Daniel Bravo Andreu and SMA Europe. Dr. Desguerre has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Has received consultancy fees from AveXis, Inc., Biogen, BioMarin, and PTC Therapeutics. Quijano-Roy. Dr. Quijano-Roy has nothing to disclose. Dr. Saito has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for Biogen, Roche/Chugai and Novartis Pharma; Consultant for AveXis.. Dr. Saito has received research support from Research funding from Biogen, Roche/Chugai and AveXis.. Dr. Droege has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AveXis, Inc., a Novartis company. Dr. Dabbous has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AveXis, Inc., a Novartis company. Dr. Shah has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of AveXis, Inc., a Novartis company, and may own Novartis stock or other equities. Dr. Farid has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AveXis Inc.Dr. Anderson has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with United Biosciences Corporation. Dr. Anderson has received research support from Portola Pharmaceuticals. Dr. Finkel has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisor fees from Biogen and Ionis Pharmaceuticals, Inc. during ENDEAR and CHERISH; advisor to AveXis, Novartis, and Roche; data safety monitoring board for the AveXis AVX-101 Phase 1 gene transfer study and Roche Moonfish Phase 1b study; advisory capacit. Dr. Finkel has received compensation for serving on the Board of Directors of Royalty payments from Children’s Hospital of Philadelphia for licensing fees obtained for use of the CHOP INTEND motor function scale. Dr. Finkel has received royalty, license fees, or contractual rights payments from Royalty payments from Children’s Hospital of Philadelphia for licensing fees obtained for use of the CHOP INTEND motor function scale. Dr. Finkel has received research support from grants from Biogen and Ionis Pharmaceuticals, Inc. during ENDEAR and CHERISH; grants from AveXis, Cytokinetics, and Roche." @default.
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