Duchenne muscular dystrophy (DMD) is a rapidly progressive X-linked disorder characterized by muscle weakening responsible for the loss of ambulation around age 12. One if limiting factor clinical development sensitivity and reliability current outcomes. Recently, 95th centile stride velocity (SV95C) was qualified European medicines agency as valid secondary outcome trials in subjects with DMD....

Dynamic mode decomposition (DMD) has become synonymous with the Koopman operator, where continuous time dynamics are discretized and examined using (i.e. composition) operators. Using newly introduced “occupation kernels,” present manuscript develops an approach to DMD that treats directly through Liouville operator. This outlines technical theoretical differences between Koopman-based for disc...

duchene and becker muscular dystrophy (dmd and bmd) are x-linked conditions that result from a defect in the dystrophin gene located on xp21. dmd is the most frequent neuromuscular disease in humans (1/3500 male newborns). in approximately 65% of dmd and bmd patients, deletions in the dystrophin gene have been identified as the molecular determinant. population-based variations in frequency and...

Background Duchenne Muscular Dystrophy (DMD), a lethal X-linked skeletal and cardiac myopathy, affects 1/3500 males[1-2]. MRI studies have shown occult ventricular dysfunction and myocardial fibrosis in DMD patients. Previously we used the Full Width of Half Maximum (FWHM) of T2 distribution in LV to quantify the myocardial structural heterogeneity in DMD patients. In DMD subject groups, we sho...

OBJECTIVE As the multiple sclerosis (MS) disease-modifying drug (DMD) treatment options have expanded to include oral therapies, it is important to understand whether route of administration is associated with DMD adherence. The objective of this study was to compare adherence to DMDs in patients with MS newly initiating treatment with a self-injectable versus an oral DMD. METHODS This retros...

Duchenne muscular dystrophy (DMD) is a common X-linked recessive disease of muscle degeneration and death. In order to provide accurate and reliable genetic counseling and prenatal diagnosis, we screened DMD mutations in a cohort of 119 Chinese patients using multiplex ligation-dependent probe amplification (MLPA) and denaturing high performance liquid chromatography (DHPLC) followed by Sanger ...

Duchenne muscular dystrophy (DMD) is a progressive and fatal muscle degenerating disease caused by a dystrophin deficiency. Effective suppression of the primary pathology observed in DMD is critical for treatment. Patient-derived human induced pluripotent stem cells (hiPSCs) are a promising tool for drug discovery. Here, we report an in vitro evaluation system for a DMD therapy using hiPSCs tha...

Duchenne muscular dystrophy (DMD) is a chronic, progressive pediatric disease that affects both muscle and brain. The objectives of the study were to examine parent reported behavior in children with DMD, investigate the influence of chronic illness, intellectual ability and etiology on behavior, and determine whether a specific behavioral profile is associated with DMD. Parental ratings of boy...

In a pilot neonatal screening programme for Duchenne muscular dystrophy (DMD) conducted in the Canadian province of Manitoba, a cohort of eight affected males was identified between 1 January 1986 and 31 December 1989. Demographic information, knowledge of DMD, reproductive outcome, and attitudes to prenatal diagnosis and neonatal screening for DMD were obtained through questionnaires distribut...