Treatment with l-citrulline and metformin in Duchenne muscular dystrophy: study protocol for a single-centre, randomised, placebo-controlled trial

P Hafner, U Bonati, D Rubino, V Gocheva, T Zumbrunn… - Trials, 2016 - Springer
Background Duchenne muscular dystrophy (DMD) is an X-linked recessive disease that
affects 1 in 3500–6000 male births. Despite broad research aiming to improve muscle …

Effect of combination l-citrulline and metformin treatment on motor function in patients with Duchenne muscular dystrophy: a randomized clinical trial

P Hafner, U Bonati, A Klein, D Rubino… - JAMA network …, 2019 - jamanetwork.com
Importance Nitric oxide precursors, such as the amino acidl-arginine and the biguanide
antidiabetic drug metformin, have been associated with metabolism and muscle function in …

Improved muscle function in Duchenne muscular dystrophy through L-arginine and metformin: an investigator-initiated, open-label, single-center, proof-of-concept …

P Hafner, U Bonati, B Erne, M Schmid, D Rubino… - PloS one, 2016 - journals.plos.org
Altered neuronal nitric oxide synthase function in Duchenne muscular dystrophy leads to
impaired mitochondrial function which is thought to be one cause of muscle damage in this …

Measurements of motor function and other clinical outcome parameters in ambulant children with Duchenne muscular dystrophy

S Nagy, S Schmidt, P Hafner, A Klein… - JoVE (Journal of …, 2019 - jove.com
While the number of new treatment options tested in patients with Duchenne muscular
dystrophy (DMD) is increasing, there is still no defining of the most reliable assessments …

Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy

G Ricci, L Bello, F Torri, E Schirinzi, E Pegoraro… - Neurological …, 2022 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastatingly severe genetic muscle
disease characterized by childhood-onset muscle weakness, leading to loss of motor …

[HTML][HTML] Emerging drugs for Duchenne muscular dystrophy

F Shawi, C Perras, M Severn - 2018 - europepmc.org
Emerging Drugs for Duchenne Muscular Dystrophy - Abstract - Europe PMC Sign in | Create
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[HTML][HTML] Current pharmacological strategies for Duchenne muscular dystrophy

S Yao, Z Chen, Y Yu, N Zhang, H Jiang… - Frontiers in Cell and …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …

Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis

S Schmidt, P Hafner, A Klein, D Rubino-Nacht… - Neuromuscular …, 2018 - Elsevier
The development of new therapeutic agents for the treatment of Duchenne muscular
dystrophy has put a focus on defining outcome measures most sensitive to capture treatment …

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 …

Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials

A Schreiber, S Brochard, P Rippert… - … Medicine & Child …, 2018 - Wiley Online Library
Aim To monitor the evolution of the motor function of ambulatory patients with Duchenne
muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with …