Results 151 to 160 of about 106,524 (297)

Interpretation of "Diagnosis and management of Duchenne muscular dystrophy: a guide for families (2011 version)"

open access: yesChinese Journal of Contemporary Neurology and Neurosurgery, 2015
The guideline "Diagnosis and management of Duchenne muscular dystrophy" was supported by a 3-year-long project guided by US Centers for Disease Control and Prevention (CDC), in collaboration with patient advocacy groups [Muscular Dystrophy Association ...
Xi-hua LI
doaj  

Muscular Dystrophy (Duchenne) in a Girl with Turner's Syndrome

open access: yesJournal of Medical Genetics, 1965
P. Ferrier, F. Bamatter, D. Klein
semanticscholar   +1 more source

Estrogen‐Related Receptor Alpha Promotes Skeletal Muscle Regeneration and Mitigates Muscular Dystrophy

open access: yesThe FASEB Journal, Volume 39, Issue 19, 15 October 2025.
ERRα promotes muscle regeneration. ERRα drives angiogenic and mitochondrial metabolic gene program in proliferating and differentiating myogenic cells. ERRα also induces myogenic factor genes such as MyoG. Through these pathways ERRα promotes muscle regeneration in the skeletal muscle in acute injury and chronic myopathy.
Thi Thu Hao Nguyen   +8 more
wiley   +1 more source

Aggravation of Cardiovascular and Respiratory Decline in Advanced Duchenne Muscular Dystrophy Complicated by Dilated Cardiomyopathy – Case Study and Review of Literature

open access: yesJournal of Education, Health and Sport
Introduction: Duchenne muscular dystrophy is a genetic X-linked recessive disorder. This condition is characterized by progressive loss of muscle tissue. Thus, it results in deterioration and inability to perform basic motor skills such as independent
Iwona Welian-Polus   +9 more
doaj   +1 more source

Remaining Burden of Spinal Muscular Atrophy Among Treated Patients: A Survey of Patients and Caregivers

open access: yesAnnals of Clinical and Translational Neurology, Volume 12, Issue 10, Page 2020-2035, October 2025.
ABSTRACT Objective Spinal muscular atrophy (SMA) significantly impacts motor function. This study aimed to assess the persistent burden and unmet needs among currently treated patients with SMA and their caregivers. Methods Two complementary web‐based surveys were distributed in August 2024 among patients with SMA and their caregivers.
Julie A. Parsons   +8 more
wiley   +1 more source

Efficacy and safety of Viltepso® in Duchenne muscular dystrophy: review of clinical studies

open access: yesРусский журнал детской неврологии
The author presents a literature review on the safety and efficacy of antisense oligonucleotides in the treatment of Duchenne muscular dystrophy using the exon skipping method using Viltepso® (viltolarsen), the only drug of this class registered in ...
V. M. Suslov, D. I. Rudenko
doaj   +1 more source

Corticosteroid therapy in Duchenne muscular dystrophy: Management and new insights

open access: yes
Developmental Medicine &Child Neurology, EarlyView.
Claudia Brogna, Eugenio Mercuri
wiley   +1 more source

Discovery and Treatment of Action Potential‐Independent Myotonia in Hyperkalemic Periodic Paralysis

open access: yesAnnals of Clinical and Translational Neurology, Volume 12, Issue 10, Page 2056-2067, October 2025.
ABSTRACT Objective Hyperkalemic periodic paralysis (hyperKPP) is characterized by attacks of transient weakness. A subset of hyperKPP patients suffers from transient involuntary contraction of muscle (myotonia). The goal of this study was to determine mechanisms causing myotonia in hyperKPP.
Chris Dupont   +4 more
wiley   +1 more source

Digital Activity Markers in Chronic Inflammatory Demyelinating Polyneuropathy

open access: yesAnnals of Clinical and Translational Neurology, Volume 12, Issue 10, Page 2045-2055, October 2025.
ABSTRACT Objective To evaluate the utility of smartwatch and smartphone‐based activity metrics for assessing disease severity and quality of life in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Methods In the electronic monitoring of disease activity in patients with CIDP (EMDA‐CIDP) trial, we performed a prospective ...
Lars Masanneck   +15 more
wiley   +1 more source

Biglycan : a multivalent proteoglycan providing structure and signals [PDF]

open access: yes, 2013
Research over the past few years has provided fascinating results indicating that biglycan, besides being a ubiquitous structural component of the extracellular matrix (ECM), may act as a signaling molecule.
Năstase, Mădălina-Viviana   +2 more
core  

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