Duchenne muscular dystrophy

D Duan, N Goemans, S Takeda, E Mercuri… - Nature Reviews …, 2021 - nature.com
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 …

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 …

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 …

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

N Wein, TA Vetter, A Vulin, TR Simmons… - … Therapy-Methods & …, 2022 - cell.com
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 …

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 …

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 …

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 …

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 …

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 …

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) …