Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

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 …

Role of L-arginine in nitric oxide synthesis and health in humans

G Wu, CJ Meininger, CJ McNeal, FW Bazer… - Amino acids in nutrition …, 2021 - Springer
As a functional amino acid (AA), L-arginine (Arg) serves not only as a building block of
protein but also as an essential substrate for the synthesis of nitric oxide (NO), creatine …

The journey of metformin from glycaemic control to mTOR inhibition and the suppression of tumour growth

S Amin, A Lux, F O'Callaghan - British journal of clinical …, 2019 - Wiley Online Library
Our knowledge of the effect of metformin on human health is increasing. In addition to its
ability to improve the control of hyperglycaemia, metformin has been shown to reduce the …

BETs inhibition attenuates oxidative stress and preserves muscle integrity in Duchenne muscular dystrophy

M Segatto, R Szokoll, R Fittipaldi, C Bottino… - Nature …, 2020 - nature.com
Duchenne muscular dystrophy (DMD) affects 1 in 3500 live male births. To date, there is no
effective cure for DMD, and the identification of novel molecular targets involved in disease …

AMPK is mitochondrial medicine for neuromuscular disorders

AI Mikhail, SY Ng, SR Mattina, V Ljubicic - Trends in Molecular Medicine, 2023 - cell.com
Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and spinal
muscular atrophy (SMA) are the most prevalent neuromuscular disorders (NMDs) in children …

Advances in the treatment of Duchenne muscular dystrophy: new and emerging pharmacotherapies

AM Reinig, S Mirzaei, DJ Berlau - … : The Journal of Human …, 2017 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disease that primarily
affects young males. Patients with DMD are unable to produce dystrophin, a crucial protein …

A brief review of Duchenne muscular dystrophy treatment options, with an emphasis on two novel strategies

A Heydemann, M Siemionow - Biomedicines, 2023 - mdpi.com
Despite the full cloning of the Dystrophin cDNA 35 years ago, no effective treatment exists
for the Duchenne Muscular Dystrophy (DMD) patients who have a mutation in this gene …

Metformin: new applications for an old drug

A Naseri, S Sanaie, S Hamzehzadeh… - Journal of Basic and …, 2023 - degruyter.com
Metformin is a biguanide, evolved as one of the most widely used medicines. The
applications of this component include but are not limited to reducing blood glucose, weight …

[HTML][HTML] Metformin reverses the enhanced myocardial SR/ER–mitochondria interaction and impaired complex I-driven respiration in dystrophin-deficient mice

C Angebault, M Panel, M Lacôte, J Rieusset… - Frontiers in Cell and …, 2021 - frontiersin.org
Besides skeletal muscle dysfunction, Duchenne muscular dystrophy (DMD) exhibits a
progressive cardiomyopathy characterized by an impaired calcium (Ca2+) homeostasis and …