Cargando…

Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada

BACKGROUND: Spinal muscular atrophy (SMA) is a rare neurodegenerative disease characterized by progressive muscular weakness, which occurs in one in 6,000 to 10,000 live births. The burden of SMA on Canadian patients and caregivers is not known. OBJECTIVE: To characterize the burden of SMA in Canada...

Descripción completa

Detalles Bibliográficos
Autores principales:	McMillan, H.J., Gerber, B., Cowling, T., Khuu, W., Mayer, M., Wu, J.W., Maturi, B., Klein-Panneton, K., Cabalteja, C., Lochmüller, H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2021
Materias:	Research Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8385498/ https://www.ncbi.nlm.nih.gov/pubmed/33749617 http://dx.doi.org/10.3233/JND-200610

Ejemplares similares

Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers
por: Sutherland, C. Simone, et al.
Publicado: (2021)

Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
por: Landfeldt, Erik, et al.
Publicado: (2021)

Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort
por: Jones, Cynthia C., et al.
Publicado: (2020)

Eighteen Years with Spinal Muscular Atrophy (SMA) Type 1
por: Valencia, Borja, et al.
Publicado: (2013)

Measuring Fatigue and Fatigability in Spinal Muscular Atrophy (SMA): Challenges and Opportunities
por: Rodriguez-Torres, Rafael S., et al.
Publicado: (2023)

An Evaluation of a Continuing Education Program for Family Caregivers of Ventilator-Dependent Children with Spinal Muscular Atrophy (SMA)
por: Boroughs, Deborah S.
Publicado: (2017)

SMA-MAP: A Plasma Protein Panel for Spinal Muscular Atrophy
por: Kobayashi, Dione T., et al.
Publicado: (2013)

A call to introduce newborn screening for spinal muscular atrophy (SMA) in Scotland
por: Gillingwater, Thomas H., et al.
Publicado: (2022)

Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study
por: Vázquez-Costa, Juan F., et al.
Publicado: (2022)

Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)
por: Paracha, Noman, et al.
Publicado: (2021)

Alambres musculares (SMA) /
por: Hernández Ramírez, Edgar
Publicado: (2001)

Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment
por: Monnette, Alisha, et al.
Publicado: (2021)

Longitudinal prospective cohort study to assess peripheral motor function with extensive electrophysiological techniques in patients with Spinal Muscular Atrophy (SMA): the SMA Motor Map protocol
por: Ros, Leandra A. A., et al.
Publicado: (2023)

Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse
por: McGovern, Vicki L., et al.
Publicado: (2015)

Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)
por: Zanetta, Chiara, et al.
Publicado: (2014)

Spontaneous Breathing Pattern as Respiratory Functional Outcome in Children with Spinal Muscular Atrophy (SMA)
por: LoMauro, A., et al.
Publicado: (2016)

Therapeutic decisions under uncertainty for spinal muscular atrophy: The DECISIONS-SMA study protocol
por: Saposnik, Gustavo, et al.
Publicado: (2022)

Correction to: Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)
por: Paracha, Noman, et al.
Publicado: (2022)

Candidate Proteins, Metabolites and Transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study
por: Finkel, Richard S., et al.
Publicado: (2012)

The Cure SMA Membership Surveys: Highlights of Key Demographic and Clinical Characteristics of Individuals with Spinal Muscular Atrophy
por: Belter, Lisa, et al.
Publicado: (2021)

Biomarker for Spinal Muscular Atrophy: Expression of SMN in Peripheral Blood of SMA Patients and Healthy Controls
por: Czech, Christian, et al.
Publicado: (2015)

The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs
por: Hensel, Niko, et al.
Publicado: (2020)

Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen
por: Wurster, Claudia D., et al.
Publicado: (2019)

Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review
por: Chong, Li Chuin, et al.
Publicado: (2021)

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1
por: Boursange, Sophie, et al.
Publicado: (2022)

The Burden of Spinal Muscular Atrophy on Informal Caregivers
por: Aranda-Reneo, Isaac, et al.
Publicado: (2020)

Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study
por: Crawford, Thomas O., et al.
Publicado: (2012)

SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe
por: Kayadjanian, Nathalie, et al.
Publicado: (2013)

Association of type IV spinal muscular atrophy (SMA) with myoclonic epilepsy within a single family
por: Liyanage, Damith S, et al.
Publicado: (2014)

Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool
por: Ramsey, Danielle, et al.
Publicado: (2017)

Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain
por: López-Bastida, Julio, et al.
Publicado: (2017)

Executive function is inversely correlated with physical function: the cognitive profile of adult Spinal Muscular Atrophy (SMA)
por: Mix, Lucas, et al.
Publicado: (2021)

Changes in Ventilatory Support Requirements of Spinal Muscular Atrophy (SMA) Patients Post Gene-Based Therapies
por: Panagiotou, Panagiota, et al.
Publicado: (2022)

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)
por: Ogbonmide, Tolu, et al.
Publicado: (2023)

Recent Progress in the Identification of the X-Linked Infantile Spinal Muscular Atrophy (Xl-SMA) Gene: Implications for Neuronal Apoptosis
por: Baumbach, L. L., et al.
Publicado: (2001)

Design of a Non-Interventional Study to Validate a Set of Patient- and Caregiver-Oriented Measurements to Assess Health Outcomes in Spinal Muscular Atrophy (SMA-TOOL Study)
por: Madruga-Garrido, Marcos, et al.
Publicado: (2021)

Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study
por: Annoussamy, Mélanie, et al.
Publicado: (2020)

Evaluation of putative CSF biomarkers in paediatric spinal muscular atrophy (SMA) patients before and during treatment with nusinersen
por: Johannsen, Jessika, et al.
Publicado: (2021)

Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study
Publicado: (2021)

Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence
por: Hjartarson, Helgi Thor, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_7e1udm5vcbnols7hmpk82rlmlb