[HTML][HTML] Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
S Kourakis, CA Timpani, DG Campelj, P Hafner… - Orphanet Journal of …, 2021 - Springer
Background Pharmacological corticosteroid therapy is the standard of care in Duchenne
Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression …
Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression …
[HTML][HTML] Targeting Nrf2 for the treatment of Duchenne muscular dystrophy
Imbalances in redox homeostasis can result in oxidative stress, which is implicated in
various pathological conditions including the fatal neuromuscular disease Duchenne …
various pathological conditions including the fatal neuromuscular disease Duchenne …
Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy
A Moretti, L Fonteyne, F Giesert, P Hoppmann… - Nature medicine, 2020 - nature.com
Frameshift mutations in the DMD gene, encoding dystrophin, cause Duchenne muscular
dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene …
dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene …
Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy
M Chesshyre, D Ridout, Y Hashimoto… - Journal of Cachexia …, 2022 - Wiley Online Library
Background Duchenne muscular dystrophy (DMD) is caused by DMD mutations leading to
dystrophin loss. Full‐length Dp427 is the primary dystrophin isoform expressed in muscle …
dystrophin loss. Full‐length Dp427 is the primary dystrophin isoform expressed in muscle …
[HTML][HTML] Inhibiting the inflammasome with MCC950 counteracts muscle pyroptosis and improves Duchenne muscular dystrophy
N Dubuisson, MA Davis-López de Carrizosa… - Frontiers in …, 2022 - frontiersin.org
Background Duchenne muscular dystrophy (DMD) is the most common inherited human
myopathy. Typically, the secondary process involving severe inflammation and necrosis …
myopathy. Typically, the secondary process involving severe inflammation and necrosis …
AdipoRon, a new therapeutic prospect for Duchenne muscular dystrophy
M Abou‐Samra, CM Selvais… - Journal of cachexia …, 2020 - Wiley Online Library
Abstract Background Adiponectin (ApN) is a hormone known to exhibit insulin‐sensitizing,
fat‐burning, and anti‐inflammatory properties in several tissues, including the skeletal …
fat‐burning, and anti‐inflammatory properties in several tissues, including the skeletal …
[HTML][HTML] De-duplicating patient records from three independent data sources reveals the incidence of rare neuromuscular disorders in Germany
K König, A Pechmann, S Thiele, MC Walter… - Orphanet journal of rare …, 2019 - Springer
Background Estimation of incidence in rare diseases is often challenging due to unspecific
and incomplete coding and recording systems. Patient-and health care provider-driven data …
and incomplete coding and recording systems. Patient-and health care provider-driven data …
[HTML][HTML] Isolation and Characterization of Primary DMD Pig Muscle Cells as an In Vitro Model for Preclinical Research on Duchenne Muscular Dystrophy
T Donandt, S Hintze, S Krause, E Wolf, B Schoser… - Life, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is the most frequent genetic myopathy in childhood
and leads to progressive muscle atrophy, weakness, and premature death. So far, there is …
and leads to progressive muscle atrophy, weakness, and premature death. So far, there is …
[HTML][HTML] Haplotype-Based Noninvasive Prenatal Diagnosis of 21 Families With Duchenne Muscular Dystrophy: Real-World Clinical Data in China
L Kong, S Li, Z Zhao, J Feng, G Chen, L Liu… - Frontiers in …, 2021 - frontiersin.org
Noninvasive prenatal diagnosis (NIPD) of single-gene disorders has recently become the
focus of clinical laboratories. However, reports on the clinical application of NIPD of …
focus of clinical laboratories. However, reports on the clinical application of NIPD of …
Aldehyde dehydrogenases contribute to skeletal muscle homeostasis in healthy, aging, and Duchenne muscular dystrophy patients
J Etienne, P Joanne, C Catelain… - Journal of cachexia …, 2020 - Wiley Online Library
Abstract Background Aldehyde dehydrogenases (ALDHs) are key players in cell survival,
protection, and differentiation via the metabolism and detoxification of aldehydes. ALDH …
protection, and differentiation via the metabolism and detoxification of aldehydes. ALDH …