S12 Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE-US)

Day, John W.; Finkel, R.; Connolly, AM; Darras, B.; Iannaccone, ST; Kuntz, N.; Peña, Ldm; Smith, EC; Chiriboga, CA; Crawford, T. B. B.; Shieh, PB; Kwon, J.M.; Zaidman, C.; Schultz, Megan L.; Kausar, Imran; Chand, Deepa H.; Tauscher-Wisniewski, Sitra; Ouyang, Haojun; Macek, Thomas A.; Mendell, J. R.

doi:10.1136/thorax-2020-btsabstracts.18

Cited by 2 publications

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“…Open-label trials showed the safety and efficacy of this approach. 83,112 Similar to pre-clinical studies, the timing of delivery was essential, with early treatment of symptomatic infants before 6 months or treatment of asymptomatic infants showing the best neurological outcomes. 83 This AAV9-mediated SMN1 gene therapy (known as Zolgensma or onasemnogene abeparvovec-xioi) was approved by the FDA in 2019.…”

Section: Reviewmentioning

confidence: 99%

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

et al. 2022

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Section: Reviewmentioning

confidence: 99%

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

et al. 2022

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“…Several successful clinical trials (CT) have been conducted over recent years which have confirmed the safety and efficacy of nusinersen, an intrathecally administered antisense oligonucleotide in SMA Types 1 and 2, [4,5], onasmnogene abeparvovec, an AAV9-mediated gene transfer in Type 1 [6,7] and risdiplam, an oral compound [8]. A review of currently available data on new treatments was published by Ramdas & Servais [9].…”

Section: Introductionmentioning

confidence: 99%

Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative

Tizzano

Christie-Brown²,

Baranello

et al. 2022

Journal of Neuromuscular Diseases

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Several successful clinical trials have been conducted in spinal muscular atrophy (SMA) over recent years which have led to the approval of splicing modifiers and gene transfer therapies. With an increasing number of other agents progressing through pre-clinical and clinical development, increasing worldwide clinical trial readiness is becoming essential. SMA Europe initiated a clinical trial readiness project, which included the development of a pilot face-to-face educational-training initiative for clinical specialists and physiotherapists involved in SMA, with an emphasis on the patient perspective. Participants were selected through two surveys and, ahead of the meeting, a mock protocol with specific questions was provided. The initiative involved a series of presentations, role-play and interactive exercises. We describe here our experience and evaluation of this educational-training initiative, emphasising scientific aspects, psychosocial implications and level of satisfaction. From a participant, patient and industry perspective, such training was considered successful and met the objective, which was to improve clinical trial readiness in emerging sites. Resource planning, ethical considerations and communication with patients were identified as three important topics for future training. This initiative highlights the need to develop a training programme to achieve clinical trial readiness across Europe and showcases a collaborative effort with different stakeholders, clinicians, patient advocacy groups and sponsors to address an important issue.

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S12 Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE-US)

Cited by 2 publications

References 0 publications

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative

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