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Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt.
Value Health Reg Issues
November 2023
Clinical Pharmacy Department, Faculty of Pharmacy, Cairo University, Cairo, Egypt.
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 but are expected to reach the market soon. This study describes 2 cost-utility models comparing the drugs with the standard of care.
View Article and Find Full Text PDFEteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia.
Case Rep Neurol
October 2022
Department of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.
Eteplirsen is an antisense oligonucleotide used in the treatment of Duchenne muscular dystrophy (DMD). The safety of eteplirsen use in individuals with rare comorbid conditions is not known. We present the case of a 4-year-old boy with a DMD exon deletion amenable to treatment with eteplirsen and comorbid sickle cell anemia.
View Article and Find Full Text PDFTranscript-Targeted Therapy Based on RNA Interference and Antisense Oligonucleotides: Current Applications and Novel Molecular Targets.
Int J Mol Sci
August 2022
Section of Medical Biochemistry, Department of Biomedical and Biotechnological Sciences, University of Catania, 95123 Catania, Italy.
The development of novel target therapies based on the use of RNA interference (RNAi) and antisense oligonucleotides (ASOs) is growing in an exponential way, challenging the chance for the treatment of the genetic diseases and cancer by hitting selectively targeted RNA in a sequence-dependent manner. Multiple opportunities are taking shape, able to remove defective protein by silencing RNA (e.g.
View Article and Find Full Text PDFComparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.
J Neuromuscul Dis
December 2021
Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Background: Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by DMD gene mutations. A relationship between exon skipping and dystrophin production in exon 51-amenable patients treated with eteplirsen (EXONDYS 51®) is established. Once-weekly eteplirsen significantly increased dystrophin, with slower decline in ambulatory function compared to baseline.
View Article and Find Full Text PDFYear in review 2019: Neuromuscular diseases.
Pediatr Pulmonol
October 2020
Department of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, Ohio.
Neuromuscular cardiopulmonary medicine is entering a new and exciting phase, with studies that assess the respiratory effect of emerging genetic and molecular therapies. In this year's neuromuscular Year in Review, we focus on Duchenne muscular dystrophy (DMD), reviewing studies that evaluate the respiratory effect of eteplirsen, the cardiopulmonary effects of ataluren, and a study comparing the use of spironolactone with eplerenone for the treatment of DMD-related cardiomyopathy.
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