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 …

[HTML][HTML] 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 …

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 …

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

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 …

Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial

M Guglieri, PR Clemens, SJ Perlman, EC Smith… - JAMA …, 2022 - jamanetwork.com
Importance Corticosteroidal anti-inflammatory drugs are widely prescribed but long-term use
shows adverse effects that detract from patient quality of life. Objective To determine if …

Effect of different corticosteroid dosing regimens on clinical outcomes in boys with Duchenne muscular dystrophy: a randomized clinical trial

M Guglieri, K Bushby, MP McDermott, KA Hart, R Tawil… - Jama, 2022 - jamanetwork.com
Importance Corticosteroids improve strength and function in boys with Duchenne muscular
dystrophy. However, there is uncertainty regarding the optimum regimen and dosage …

Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: a meta‐analysis of disease progression rates in recent multicenter clinical trials

CM McDonald, G Sajeev, Z Yao, E McDonnell… - Muscle & …, 2020 - Wiley Online Library
Introduction In this study we characterized disease progression over 48 weeks among boys
receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent …

Outcome measures in Duchenne muscular dystrophy: sensitivity to change, clinical meaningfulness, and implications for clinical trials.

J Domingos, F Muntoni - Developmental Medicine and Child …, 2017 - europepmc.org
Outcome measures in Duchenne muscular dystrophy: sensitivity to change, clinical
meaningfulness, and implications for clinical trials. - Abstract - Europe PMC Sign in | Create an …

[HTML][HTML] Can quantitative muscle strength and functional motor ability differentiate the influence of age and corticosteroids in ambulatory boys with Duchenne muscular …

C Buckon, S Sienko, A Bagley, M Sison-Williamson… - PLoS …, 2016 - ncbi.nlm.nih.gov
Background: In the absence of a curative treatment for Duchenne Muscular Dystrophy
(DMD), corticosteroid therapy (prednisone, deflazacort) has been adopted as the standard of …

Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial

EAC Beenakker, JM Fock, MJ Van Tol… - Archives of …, 2005 - jamanetwork.com
Background Prednisone treatment is used to prolong ambulation in patients with Duchenne
muscular dystrophy (DMD). However, since severe adverse effects often accompany …