Duchenne muscular dystrophy
Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …
to difficulties with movement and, eventually, to the need for assisted ventilation and …
Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future
A Łoboda, J Dulak - Pharmacological Reports, 2020 - Springer
Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …
childhood disorder that causes progressive muscle weakness and degeneration and results …
Protein biomarker quantification by immunoaffinity liquid chromatography–tandem mass spectrometry: current state and future vision
H Neubert, CM Shuford, TV Olah, F Garofolo… - Clinical …, 2020 - academic.oup.com
Immunoaffinity–mass spectrometry (IA-MS) is an emerging analytical genre with several
advantages for profiling and determination of protein biomarkers. Because IA-MS combines …
advantages for profiling and determination of protein biomarkers. Because IA-MS combines …
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD
reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an …
reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an …
The future of exon skipping for Duchenne muscular dystrophy
A Aartsma-Rus - Human Gene Therapy, 2023 - liebertpub.com
Antisense oligonucleotide (ASO)-mediated exon skipping can restore the open reading
frame of dystrophin transcripts for Duchenne muscular dystrophy (DMD) patients. This …
frame of dystrophin transcripts for Duchenne muscular dystrophy (DMD) patients. This …
Lessons learned from discontinued clinical developments in Duchenne muscular dystrophy
T Markati, L De Waele, U Schara-Schmidt… - Frontiers in …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of
functional dystrophin protein. Patients experience progressive muscle weakness …
functional dystrophin protein. Patients experience progressive muscle weakness …
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
V Himič, KE Davies - European Journal of Human Genetics, 2021 - nature.com
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
Hydrogen sulfide as a therapeutic option for the treatment of Duchenne muscular dystrophy and other muscle-related diseases
K Kaziród, M Myszka, J Dulak, A Łoboda - Cellular and Molecular Life …, 2022 - Springer
Hydrogen sulfide (H2S) has been known for years as a poisoning gas and until recently
evoked mostly negative associations. However, the discovery of its gasotransmitter functions …
evoked mostly negative associations. However, the discovery of its gasotransmitter functions …
Next generation exon 51 skipping antisense oligonucleotides for Duchenne muscular dystrophy
J van Deutekom, C Beekman, S Bijl… - nucleic acid …, 2023 - liebertpub.com
In the last two decades, antisense oligonucleotides (AONs) that induce corrective exon
skipping have matured as promising therapies aimed at tackling the dystrophin deficiency …
skipping have matured as promising therapies aimed at tackling the dystrophin deficiency …
Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After …
A Niezgoda, G Biegański, J Wachowiak… - Archivum Immunologiae …, 2023 - Springer
Duchenne muscular dystrophy (DMD) is a lethal X-linked disease caused by mutations in
the dystrophin gene, leading to muscle degeneration and wasting. Electromyography (EMG) …
the dystrophin gene, leading to muscle degeneration and wasting. Electromyography (EMG) …