[HTML][HTML] Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy

L Servais, LL Lair, AM Connolly, BJ Byrne… - International Journal of …, 2024 - mdpi.com
Spinal muscular atrophy (SMA) is a rare, genetic neurodegenerative disorder caused by
insufficient production of survival motor neuron (SMN) protein. Diminished SMN protein …

Disease Trajectories in the revised hammersmith scale in a cohort of untreated patients with spinal muscular atrophy types 2 and 3

A Wolfe, G Stimpson, D Ramsey… - Journal of …, 2024 - journals.sagepub.com
Background: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterised by
progressive motor function decline. Motor function is assessed using several functional …

Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An …

G Coratti, F Bovis, MC Pera, M Scoto… - European Journal of …, 2024 - Wiley Online Library
Background and purpose Spinal muscular atrophy (SMA) is a rare and progressive
neuromuscular disorder with varying severity levels. The aim of the study was to calculate …

[HTML][HTML] Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module

G Coratti, M Civitello, A Rohwer, E Albamonte… - Neuromuscular …, 2024 - Elsevier
The Revised upper limb module (RULM) has been increasingly used in clinical trials and in
clinical settings. The aim of this study was to use the 'shift analysis' to assess the patterns of …

[PDF][PDF] PROTOCOLO DE MEDICIÓN DEL NIVEL FUNCIONAL DE USUARIO CON ATROFIA MUSCULAR ESPINAL TIPO II Y III SOMETIDOS A TRATAMIENTO DE …

PH Lobos - Santiago, 2024 - repositorio.unab.cl
Resumen La Atrofia Muscular Espinal, es una enfermedad de causa genética autosómica
recesiva que afecta principalmente a las neuronas motoras, específicamente por …

31. Đánh giá chức năng vận động của bệnh nhân teo cơ tủy sống

NT Hằng, VC Dũng, NN Khánh - Tạp chí Nghiên cứu Y …, 2024 - tapchinghiencuuyhoc.vn
Tóm tắt Teo cơ tủy sống (spinal muscular atrophy-SMA) là bệnh lý thần kinh cơ di truyền lặn
nhiễm sắc thể thường, đặc trưng bởi yếu và teo cơ toàn thân, tiến triển nặng dần tới tàn tật …