Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
E Tambuyzer, B Vandendriessche, CP Austin… - Nature Reviews Drug …, 2020 - nature.com
Most rare diseases still lack approved treatments despite major advances in research
providing the tools to understand their molecular basis, as well as legislation providing …
providing the tools to understand their molecular basis, as well as legislation providing …
Therapeutic approaches for Duchenne muscular dystrophy
TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy
DG Allen, NP Whitehead… - Physiological …, 2016 - journals.physiology.org
Dystrophin is a long rod-shaped protein that connects the subsarcolemmal cytoskeleton to a
complex of proteins in the surface membrane (dystrophin protein complex, DPC), with further …
complex of proteins in the surface membrane (dystrophin protein complex, DPC), with further …
The pathogenesis and therapy of muscular dystrophies
Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …
explosion in the identification of the genes and their encoded proteins responsible for these …
[PDF][PDF] Muscle damage in mdx (dystrophic) mice: role of calcium and reactive oxygen species.
Muscle damage in mdx (dystrophic) mice: The role of calcium and reactive oxygen species
Page 1 Proceedings of the Australian Physiological Society (2005) 36: 111-117 http://www.aups.org.au/Proceedings/36/111-117 …
Page 1 Proceedings of the Australian Physiological Society (2005) 36: 111-117 http://www.aups.org.au/Proceedings/36/111-117 …
Dystrophic heart failure blocked by membrane sealant poloxamer
S Yasuda, DW Townsend, DE Michele, EG Favre… - Nature, 2005 - nature.com
Dystrophin deficiency causes Duchenne muscular dystrophy (DMD) in humans, an inherited
and progressive disease of striated muscle deterioration that frequently involves …
and progressive disease of striated muscle deterioration that frequently involves …
[HTML][HTML] Current status of pharmaceutical and genetic therapeutic approaches to treat DMD
C Pichavant, A Aartsma-Rus, PR Clemens, KE Davies… - Molecular Therapy, 2011 - cell.com
Duchenne muscular dystrophy (DMD) is a genetic disease affecting about one in every
3,500 boys. This X-linked pathology is due to the absence of dystrophin in muscle fibers …
3,500 boys. This X-linked pathology is due to the absence of dystrophin in muscle fibers …
Effects of stretch‐activated channel blockers on [Ca2+]i and muscle damage in the mdx mouse
EW Yeung, NP Whitehead, TM Suchyna… - The Journal of …, 2005 - Wiley Online Library
The mdx mouse lacks dystrophin and is a model of human Duchenne muscular dystrophy.
Single mdx muscle fibres were isolated and subjected to a series of stretched (eccentric) …
Single mdx muscle fibres were isolated and subjected to a series of stretched (eccentric) …
[图书][B] Handbook of laboratory animal science: Animal Models, Volume III
J Hau, SJ Schapiro, GL Van Hoosier Jr - 2004 - taylorfrancis.com
Building upon the success of the Handbook of Laboratory Animal Science and completing
Volumes I and II of the Second Edition, Handbook of Laboratory Animal Science, Second …
Volumes I and II of the Second Edition, Handbook of Laboratory Animal Science, Second …
Duchenne's muscular dystrophy: animal models used to investigate pathogenesis and develop therapeutic strategies
CA Collins, JE Morgan - International journal of experimental …, 2003 - Wiley Online Library
Duchenne's muscular dystrophy (DMD) is a lethal childhood disease caused by mutations of
the dystrophin gene, the protein product of which, dystrophin, has a vital role in maintaining …
the dystrophin gene, the protein product of which, dystrophin, has a vital role in maintaining …