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A National Spinal Muscular Atrophy Registry for Real-World Evidence
- Victoria L. Hodgkinson, Maryam Oskoui, Joshua Lounsberry, Saïd M’Dahoma, Emily Butler, Craig Campbell, Alex MacKenzie, Hugh J. McMillan, Louise Simard, Jiri Vajsar, Bernard Brais, Kristine M. Chapman, Nicolas Chrestian, Meghan Crone, Peter Dobrowolski, Susan Dojeiji, James J. Dowling, Nicolas Dupré, Angela Genge, Hernan Gonorazky, Simona Hasal, Aaron Izenberg, Wendy Johnston, Edward Leung, Hanns Lochmüller, Jean K. Mah, Alier Marerro, Rami Massie, Laura McAdam, Anna McCormick, Michel Melanson, Michelle M. Mezei, Cam-Tu E. Nguyen, Colleen O’Connell, Erin K. O’Ferrall, Gerald Pfeffer, Cecile Phan, Stephanie Plamondon, Chantal Poulin, Xavier Rodrigue, Kerri L. Schellenberg, Kathy Selby, Jordan Sheriko, Christen Shoesmith, Garth Smith, Monique Taillon, Sean Taylor, Jodi Warman Chardon, Scott Worley, Lawrence Korngut
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- Journal:
- Canadian Journal of Neurological Sciences / Volume 47 / Issue 6 / November 2020
- Published online by Cambridge University Press:
- 04 June 2020, pp. 810-815
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- Article
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Background:
Spinal muscular atrophy (SMA) is a devastating rare disease that affects individuals regardless of ethnicity, gender, and age. The first-approved disease-modifying therapy for SMA, nusinursen, was approved by Health Canada, as well as by American and European regulatory agencies following positive clinical trial outcomes. The trials were conducted in a narrow pediatric population defined by age, severity, and genotype. Broad approval of therapy necessitates close follow-up of potential rare adverse events and effectiveness in the larger real-world population.
Methods:The Canadian Neuromuscular Disease Registry (CNDR) undertook an iterative multi-stakeholder process to expand the existing SMA dataset to capture items relevant to patient outcomes in a post-marketing environment. The CNDR SMA expanded registry is a longitudinal, prospective, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials.
Results:The consensus expanded dataset includes items that address therapy effectiveness and safety and is collected in a multicenter, prospective, observational study, including SMA patients regardless of therapeutic status. The expanded dataset is aligned with global datasets to facilitate collaboration. Additionally, consensus dataset development aimed to standardize appropriate outcome measures across the network and broader Canadian community. Prospective outcome studies, data use, and analyses are independent of the funding partner.
Conclusion:Prospective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients.
8 - Mental Health
- Edited by Sarah Pollock, Kate Parkinson, Ian Cummins, University of Salford
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- Book:
- Social Work and Society
- Published by:
- Bristol University Press
- Published online:
- 17 April 2021
- Print publication:
- 27 November 2019, pp 115-131
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Summary
Introduction
This chapter provides an overview of some of the key political processes shaping mental health policy and their implications for social work practice in this setting. It begins with a historical outline of policy responses to mental distress from the era of the asylum, through the 20th-century development of, first, hospital and then community care, and into the present period characterised by increasingly market-oriented and residualised service provision and individual responsibility placed on service users. In spite of these challenges from above, there remain a number of resources of hope for mental health social work. These emerge from person-centred initiatives and collective struggles ‘from below’ by social workers and service users to develop and extend support rooted in the values of social justice. The chapter gives examples of these and concludes with a case study to illustrate implications for practice.
Historical context of service provision
Mental health social work practice is significantly influenced by the policy context in which it takes place. Mental health policy in the UK is continually evolving, and shaped by a range of legal, professional and organisational strands (Glasby and Tew, 2015) within a wider context of neoliberal ideology (see Chapter 4). In order to understand social work in mental health in the present, it is necessary to reflect on the historical processes and institutions that have shaped it. There have been four key stages in the history of mental health service provision in the UK which, we argue, continue to exert an influence on contemporary policy and practice: the custodial asylum, the biomedical hospital system, community care and the current neoliberal period. In this section we outline the first three of these historical stages of mental health policy and practice. In the following section we then describe the main contours and dynamics of the contemporary neoliberal era and its implications for mental health social work.
Custodialism and the asylum
The primary response to mental distress in Britain during the Victorian era and before would have been either the removal of the person to a small-scale private ‘madhouse’ or segregation into one of the large-scale public asylums that had emerged by the early 19th century (Porter, 1987).
Provincial Differences in the Diagnosis and Care of Amyotrophic Lateral Sclerosis
- Victoria L. Hodgkinson, Josh Lounsberry, Ario Mirian, Angela Genge, Timothy Benstead, Hannah Briemberg, Ian Grant, Walter Hader, Wendy S. Johnston, Sanjay Kalra, Gary Linassi, Rami Massie, Michel Melanson, Colleen O’Connell, Kerri Schellenberg, Christen Shoesmith, Sean Taylor, Scott Worley, Lorne Zinman, Lawrence Korngut
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- Journal:
- Canadian Journal of Neurological Sciences / Volume 45 / Issue 6 / November 2018
- Published online by Cambridge University Press:
- 15 November 2018, pp. 652-659
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Background: Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease resulting in muscle weakness, dysarthria and dysphagia, and ultimately respiratory failure leading to death. Half of the ALS patients survive less than 3 years, and 80% of the patients survive less than 5 years. Riluzole is the only approved medication in Canada with randomized controlled clinical trial evidence to slow the progression of ALS, albeit only to a modest degree. The Canadian Neuromuscular Disease Registry (CNDR) collects data on over 140 different neuromuscular diseases including ALS across ten academic institutions and 28 clinics including ten multidisciplinary ALS clinics. Methods: In this study, CNDR registry data were analyzed to examine potential differences in ALS care among provinces in time to diagnosis, riluzole and feeding tube use. Results: Significant differences were found among provinces, in time to diagnosis from symptom onset, in the use of riluzole and in feeding tube use. Conclusions: Future investigations should be undertaken to identify factors contributing to such differences, and to propose potential interventions to address the provincial differences reported.