Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy
J Burakiewicz, CDJ Sinclair, D Fischer, GA Walter… - Journal of …, 2017 - Springer
The muscular dystrophies are rare orphan diseases, characterized by progressive muscle
weakness: the most common and well known is Duchenne muscular dystrophy which affects …
weakness: the most common and well known is Duchenne muscular dystrophy which affects …
Duchenne and Becker muscular dystrophies
KM Flanigan - Neurologic clinics, 2014 - neurologic.theclinics.com
The X-linked Duchenne and Becker muscular dystrophies (DMD and BMD) are allelic
disorders occurring due to mutations in the DMD gene, which consists of 79 exons encoding …
disorders occurring due to mutations in the DMD gene, which consists of 79 exons encoding …
Development of the P erformance of the U pper L imb module for D uchenne muscular dystrophy
A Mayhew, ES Mazzone, M Eagle… - … Medicine & Child …, 2013 - Wiley Online Library
Aim An international C linical O utcomes G roup consisting of clinicians, scientists, patient
advocacy groups, and industries identified a need for a scale to measure motor performance …
advocacy groups, and industries identified a need for a scale to measure motor performance …
Assisted bicycle training delays functional deterioration in boys with Duchenne muscular dystrophy: the randomized controlled trial “no use is disuse”
M Jansen, N van Alfen, ACH Geurts… - … and neural repair, 2013 - journals.sagepub.com
Background. Physical training might delay the functional deterioration caused by disuse in
boys with Duchenne muscular dystrophy (DMD). The “No Use Is Disuse” study is the first …
boys with Duchenne muscular dystrophy (DMD). The “No Use Is Disuse” study is the first …
Motor and respiratory heterogeneity in Duchenne patients: implication for clinical trials
V Humbertclaude, D Hamroun, K Bezzou… - European Journal of …, 2012 - Elsevier
AIMS: Our objective was to clarify the clinical heterogeneity in Duchenne muscular
dystrophy (DMD). METHODS: The French dystrophinopathy database provided clinical …
dystrophy (DMD). METHODS: The French dystrophinopathy database provided clinical …
Modeling disease trajectory in Duchenne muscular dystrophy
Objective To quantify disease progression in individuals with Duchenne muscular dystrophy
(DMD) using magnetic resonance biomarkers of leg muscles. Methods MRI and magnetic …
(DMD) using magnetic resonance biomarkers of leg muscles. Methods MRI and magnetic …
Quantitative muscle MRI: a powerful surrogate outcome measure in Duchenne muscular dystrophy
U Bonati, P Hafner, S Schädelin, M Schmid… - Neuromuscular …, 2015 - Elsevier
In muscular dystrophies quantitative muscle MRI (qMRI) detects disease progression more
sensitively than clinical scores. This prospective one year observational study compared …
sensitively than clinical scores. This prospective one year observational study compared …
[HTML][HTML] Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings
K Bushby, E Connor - Clinical investigation, 2011 - ncbi.nlm.nih.gov
Abstract In June 2010, 25 representatives from Europe and the US met in Washington, DC,
USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the …
USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the …
[HTML][HTML] Prevalence and distribution of late gadolinium enhancement in a large population of patients with Duchenne muscular dystrophy: effect of age and left …
KN Hor, MD Taylor, HR Al-Khalidi, LH Cripe… - Journal of …, 2013 - Elsevier
Background Duchenne muscular dystrophy (DMD), an X-linked disorder affects
approximately 1 in 5000 males, is universally associated with heart disease. We previously …
approximately 1 in 5000 males, is universally associated with heart disease. We previously …
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
BC Henzi, S Schmidt, S Nagy, D Rubino-Nacht… - The Lancet …, 2023 - thelancet.com
Background Drug repurposing could provide novel treatment options for Duchenne
muscular dystrophy. Because tamoxifen—an oestrogen receptor regulator—reduced signs …
muscular dystrophy. Because tamoxifen—an oestrogen receptor regulator—reduced signs …