Emerging therapies for Duchenne muscular dystrophy

T Markati, M Oskoui, MA Farrar, T Duong… - The Lancet …, 2022 - thelancet.com
Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

[HTML][HTML] Ginseng and ginsenosides: Therapeutic potential for sarcopenia

W Zha, Y Sun, W Gong, L Li, W Kim, H Li - Biomedicine & Pharmacotherapy, 2022 - Elsevier
Sarcopenia is a progressive syndrome that results in a decline in skeletal muscle mass and
strength. This affects patients' quality of life and increases the risk of falls and fractures …

[HTML][HTML] The failed clinical story of myostatin inhibitors against Duchenne muscular dystrophy: exploring the biology behind the battle

E Rybalka, CA Timpani, DA Debruin, RM Bagaric… - Cells, 2020 - mdpi.com
Myostatin inhibition therapy has held much promise for the treatment of muscle wasting
disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy …

[HTML][HTML] Delivery of oligonucleotides: efficiency with lipid conjugation and clinical outcome

P Tran, T Weldemichael, Z Liu, H Li - Pharmaceutics, 2022 - mdpi.com
Oligonucleotides have shifted drug discovery into a new paradigm due to their ability to
silence the genes and inhibit protein translation. Importantly, they can drug the un-druggable …

Therapeutic approaches to preserve the musculature in Duchenne Muscular Dystrophy: The importance of the secondary therapies

G Angelini, G Mura, G Messina - Experimental Cell Research, 2022 - Elsevier
Muscular dystrophies (MDs) are heterogeneous diseases, characterized by primary wasting
of skeletal muscle, which in severe cases, such as Duchenne Muscular Dystrophy (DMD) …

[HTML][HTML] MIF1 and MIF2 myostatin peptide inhibitors as potent muscle mass regulators

EJ Lee, S Shaikh, MH Baig, SY Park, JH Lim… - International Journal of …, 2022 - mdpi.com
The use of peptides as drugs has progressed over time and continues to evolve as treatment
paradigms change and new drugs are developed. Myostatin (MSTN) inhibition therapy has …

[HTML][HTML] 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 …

[HTML][HTML] Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy

SP Sherlock, J Palmer, KR Wagner, HZ Abdel-Hamid… - Scientific reports, 2022 - nature.com
We evaluated whether whole-body dual-energy X-ray absorptiometry (DXA) measures of
lean body mass can be used as biomarkers for disease progression and treatment effects in …

Association of lithocholic acid with skeletal muscle hypertrophy through TGR5-IGF-1 and skeletal muscle mass in cultured mouse myotubes, chronic liver disease rats …

Y Tamai, A Eguchi, R Shigefuku, H Kitamura… - Elife, 2022 - elifesciences.org
Background: Hepatic sarcopenia is one of many complications associated with chronic liver
disease (CLD) and has a high mortality rate; however, the liver-muscle axis is not fully …

[HTML][HTML] Deciphering myostatin's regulatory, metabolic, and developmental influence in skeletal diseases

CL Omosule, CL Phillips - Frontiers in Genetics, 2021 - frontiersin.org
Current research findings in humans and other mammalian and non-mammalian species
support the potent regulatory role of myostatin in the morphology and function of muscle as …