Cargando…

Comprehensive assessment model for patients with spinal muscular atrophy: proposal of tools for clinical practice and real-world studies

Detalles Bibliográficos
Autores principales:	Alves, Renalli Manuella Rodrigues, Van Der Linden, Vanessa, de Andrade, Lívia Barbosa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Associação Médica Brasileira 2023
Materias:	Short Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10004283/ https://www.ncbi.nlm.nih.gov/pubmed/36820780 http://dx.doi.org/10.1590/1806-9282.20221608

Ejemplares similares

Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study
por: Elman, Lauren, et al.
Publicado: (2022)

A National Spinal Muscular Atrophy Registry for Real-World Evidence
por: Hodgkinson, Victoria L., et al.
Publicado: (2020)

Brazilian version of the CHOP INTEND scale: cross-cultural adaptation and validation
por: Alves, Renalli Manuella Rodrigues, et al.
Publicado: (2023)

Real-World Data on Access to Standards of Care for People With Spinal Muscular Atrophy in the UK
por: Muni-Lofra, Robert, et al.
Publicado: (2022)

Significant healthcare burden and life cost of spinal muscular atrophy: real-world data
por: Chan, Sophelia H. S., et al.
Publicado: (2022)

Comprehensive Modeling of Spinal Muscular Atrophy in Drosophila melanogaster
por: Spring, Ashlyn M., et al.
Publicado: (2019)

Spinal muscular atrophy
por: D'Amico, Adele, et al.
Publicado: (2011)

Effectiveness of Nusinersen in Type 1, 2 and 3 Spinal Muscular Atrophy: Croatian Real-World Data
por: Belančić, Andrej, et al.
Publicado: (2023)

Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland
por: Krupa, Dominika, et al.
Publicado: (2023)

Only some patients with bulbar and spinal muscular atrophy may develop cardiac disease
por: Finsterer, Josef, et al.
Publicado: (2017)

Only some patients with bulbar and spinal muscular atrophy may develop cardiac disease
por: Finsterer, Josef, et al.
Publicado: (2017)

Subjective Sleep Quality as it Relates to Cognitive and Physical Function in Spinal Muscular Atrophy Patients
por: Wennberg, Alexandra, et al.
Publicado: (2023)

Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification
por: Proud, Crystal M., et al.
Publicado: (2023)

Nusinersen for spinal muscular atrophy
por: Wurster, Claudia D., et al.
Publicado: (2018)

Spinal Muscular Atrophy and Ependymoma
por: Albakr, Aishah, et al.
Publicado: (2017)

Risdiplam for spinal muscular atrophy
Publicado: (2022)

Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
por: Bitetti, Ilaria, et al.
Publicado: (2022)

Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical–Experimental, and Real-World Evidence
por: Crisafulli, Salvatore, et al.
Publicado: (2023)

Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy
por: Orbach, Rotem, et al.
Publicado: (2022)

Modeling Spinal Muscular Atrophy in Drosophila
por: Chang, Howard Chia-Hao, et al.
Publicado: (2008)

Carrier screening for spinal muscular atrophy
por: Prior, Thomas W.
Publicado: (2008)

Androgens in spinal and bulbar muscular atrophy
Publicado: (2012)

Spinal Muscular Atrophy: The Treatment Approved
por: Tabet, Rabih, et al.
Publicado: (2017)

Advances in spinal muscular atrophy therapeutics
por: Parente, Valeria, et al.
Publicado: (2018)

Euglycemic Ketoacidosis in Spinal Muscular Atrophy
por: Stoimenis, Dimitrios, et al.
Publicado: (2019)

Metabolic Dysfunction in Spinal Muscular Atrophy
por: Deguise, Marc-Olivier, et al.
Publicado: (2021)

Update on Biomarkers in Spinal Muscular Atrophy
por: Pino, Megan G, et al.
Publicado: (2021)

Onasemnogene abeparvovec for spinal muscular atrophy
Publicado: (2022)

Mitochondrial Dysfunction in Spinal Muscular Atrophy
por: Zilio, Eleonora, et al.
Publicado: (2022)

Editorial: Advances in spinal muscular atrophy
por: Sun, Jianli
Publicado: (2023)

Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources
por: Youn, Bora, et al.
Publicado: (2023)

Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy
por: Montes, Jacqueline, et al.
Publicado: (2019)

Impaired kidney structure and function in spinal muscular atrophy
por: Nery, Flávia C., et al.
Publicado: (2019)

Observation of the natural course of type 3 spinal muscular atrophy: data from the polish registry of spinal muscular atrophy
por: Lusakowska, Anna, et al.
Publicado: (2021)

Long-term nusinersen treatment across a wide spectrum of spinal muscular atrophy severity: a real-world experience
por: Łusakowska, Anna, 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)

Mitochondrial abnormalities in spinal and bulbar muscular atrophy
por: Ranganathan, Srikanth, et al.
Publicado: (2009)

Rapid molecular diagnosis of spinal muscular atrophy
por: Mittal, Balraj
Publicado: (2012)

Cardiac involvement in patients with Spinal Muscular Atrophies
por: Palladino, Alberto, et al.
Publicado: (2011)

Clinical features of spinal and bulbar muscular atrophy
por: Rhodes, Lindsay E., et al.
Publicado: (2009)

Cannot write session to /tmp/vufind_sessions/sess_ein1mjhgj80g9pbr4r2qnf5n5j