Physiological stress improves stem cell modeling of dystrophic cardiomyopathy

DE Fullenkamp, AB Willis, JL Curtin… - Disease Models & …, 2024 - journals.biologists.com
Heart failure contributes to Duchenne muscular dystrophy (DMD), which arises from
mutations that ablate dystrophin, rendering the plasma membrane prone to disruption …

[HTML][HTML] Wearable Inertial Devices in Duchenne Muscular Dystrophy: A Scoping Review

E Panero, R D'Alessandro, I Cavallina, C Davico… - Applied Sciences, 2023 - mdpi.com
In clinical practice and research, innovative digital technologies have been proposed for the
characterization of neuromuscular and movement disorders through objective measures …

[HTML][HTML] Gait classification for growing children with Duchenne muscular dystrophy

I Vandekerckhove, E Papageorgiou, B Hanssen… - Scientific Reports, 2024 - nature.com
Classifying gait patterns into homogeneous groups could enhance communication among
healthcare providers, clinical decision making and clinical trial designs in boys with …

[HTML][HTML] N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy

JF Boehler, KJ Brown, V Ricotti, CA Morris - Skeletal Muscle, 2024 - Springer
Background Multiple clinical trials to assess the efficacy of AAV-directed gene transfer in
participants with Duchenne muscular dystrophy (DMD) are ongoing. The success of these …

Hybrid QUS Radiomics: A Multimodal-Integrated Quantitative Ultrasound Radiomics for Assessing Ambulatory Function in Duchenne Muscular Dystrophy

D Yan, Q Li, CW Lin, JY Shieh… - IEEE Journal of …, 2023 - ieeexplore.ieee.org
Background: Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that affects
ambulatory function. Quantitative ultrasound (QUS) imaging, utilizing envelope statistics, has …

Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt

Z Shehata, A Metry, H Rabea, R El Sherif… - Value in Health …, 2023 - Elsevier
Objectives Ataluren and eteplirsen are orphan drugs that delay progression of Duchenne
muscular dystrophy in mutation-specific subgroups. They have yet to be approved in Egypt …

[HTML][HTML] Консенсус по концепции современной эффективной терапии мышечной дистрофии Дюшенна

ТА Гремякова, СБ Артемьева… - Нервно-мышечные …, 2023 - cyberleninka.ru
Мышечная дистрофия Дюшенна-генетическое орфанное нервно-мышечное
заболевание, обусловленное мутацией гена DMD, кодирующего белок дистрофин. В …

[HTML][HTML] Gain and loss of upper limb abilities in Duchenne muscular dystrophy patients: A 24-month study

G Coratti, M Pane, C Brogna, A D'Amico… - Neuromuscular …, 2024 - Elsevier
Duchenne muscular dystrophy (DMD) is a neuromuscular condition characterized by muscle
weakness. The Performance of upper limb (PUL) test is designed to evaluate upper limb …

[HTML][HTML] Proteomic strategies for blood biomarker development in rare dystrophinopathies

C Johansson - 2023 - diva-portal.org
Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are two rare
genetic disorders of the family dystrophinopathy. They are both caused by the lack of, or …

Epidemiological Study of Neuromuscular Diseases in the Varna Region

МВ Грудкова - 2023 - search.proquest.com
Наследствените неврологични заболявания представляват широка хетерогенна група
от болести, които засягат главния и гръбначния мозък, мускулите и нервите. По-голяма …