Results 151 to 160 of about 106,524 (297)
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
P. Ferrier, F. Bamatter, D. Klein
semanticscholar +1 more source
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
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
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
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
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
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
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]
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

