Detecting early signs in Duchenne muscular dystrophy: comprehensive review and diagnostic implications

E Mercuri, M Pane, G Cicala, C Brogna… - Frontiers in …, 2023 - frontiersin.org
Despite the early onset of clinical signs suggestive of Duchenne muscular dystrophy (DMD),
a diagnosis is often not made until four years of age or older, with a diagnostic delay of up to …

The complex landscape of DMD mutations: moving towards personalized medicine

F Gatto, S Benemei, G Piluso, L Bello - Frontiers in Genetics, 2024 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by
progressive muscle degeneration, with respiratory and cardiac complications, caused by …

Age, corticosteroid treatment and site of mutations affect motor functional changes in young boys with Duchenne Muscular Dystrophy

G Coratti, J Lenkowicz, G Norcia, S Lucibello… - Plos one, 2022 - journals.plos.org
The aim of this study was to establish the possible effect of age, corticosteroid treatment and
brain dystrophin involvement on motor function in young boys affected by Duchenne …

Reference curves of motor function outcomes in young steroid‐naïve males with Duchenne muscular dystrophy

J Hoskens, M Schiava, N Goemans… - … Medicine & Child …, 2024 - Wiley Online Library
Aim To investigate functional motor performance in a large cohort of young steroid‐naïve
males with Duchenne muscular dystrophy (DMD) and typically developing males, and to …

How do fine and gross motor skills develop in preschool boys with Duchenne Muscular Dystrophy?

J Hoskens, I Vandekerckhove, L De Waele… - Research in …, 2024 - Elsevier
Abstract Background Boys with Duchenne Muscular Dystrophy (DMD) experience both fine
and gross motor problems. Nowadays, early intervention focuses almost exclusively on …

[HTML][HTML] Understanding North Star Ambulatory Assessment total scores and their implications for standards of care using observational data

G Stimpson, MK James, M Guglieri, A Wolfe… - European Journal of …, 2024 - Elsevier
Abstract NorthStar Ambulatory Assessment (NSAA) total score (TS) is an ordinal scale to
evaluate disease progression and treatment response in ambulatory Duchenne Muscular …

[PDF][PDF] Verlaufsuntersuchung degenerativer Myopathien mittels Mechanographie im Vergleich zu konventionellen etablierten Funktionstestungen

F Dreier - 2024 - edoc.ub.uni-muenchen.de
1.5. 1 Klassische Funktionstestungen Der bewährteste Test zur Beurteilung der motorischen
Fähigkeiten bei Patienten mit neuromuskulären Erkrankungen ist der 6MWT. Einerseits wird …

Применение препарата аталурен для лечения пациентов с мышечной дистрофией Дюшенна в реальной клинической практике

СГ Попович, ЛМ Кузенкова, ЕВ Увакина… - … журнал имени ЛО …, 2024 - neuro-journal.ru
Аннотация Цель. Провести анализ анамнестических, клинических и параклинических
показателей у пациентов с мышечной дистрофией Дюшенна (МДД), получающих …

Multiomic investigations in neuromuscular diseases

I Dabaj - 2023 - policycommons.net
Pediatric neuromuscular disorders encompass all childhood onset diseases affecting the
muscle, the neuromuscular junction, peripheral nerve, and the anterior horn cell. The …

[PDF][PDF] Les maladies neuromusculaires d'origine génétique (expérience de l'unité de génétique du CHU Hassan II FES)

A Salma - 2022 - toubkal.imist.ma
LES MALADIES NEUROMUSCULAIRES D'ORIGINE GENETIQUE Page 1 Année 2022
Thèse N 008/22 THÈSE PRÉSENTEE ET SOUTENUE PUBLIQUEMENT LE 04/01/2022 …