Results 111 to 120 of about 182,997 (357)
Genetics and the muscular dystrophies [PDF]
Developmental Medicine & Child Neurology, 2000 Muscular dystrophy classification and diagnosis has been transformed over the 13 or so years since the cloning of the dystrophin gene (involved in Duchenne and Becker muscular dystrophy). A child or adult presenting with a muscular dystrophy can now expect a precise diagnosis.
openaire +4 more sources
Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks
Chemistry – A European Journal, EarlyView.A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan +8 more
wiley +1 more source
A Price on Volunteerism:The Public Has a Higher Duty to Accommodate Volunteers [PDF]
, 2007 This Comment first examines the issues presented in Bauer (including the holding that the Americans with Disabilities Act does not protect these volunteers from discrimination) and the court’s rationale for finding that volunteers are not protected under
Attard, Lauren
core +1 more source
Young people’s experiences using electric powered indoor-outdoor wheelchairs (EPIOCs): Potential for enhancing users’ development? [PDF]
, 2007 Purpose: To examine the experiences of severely physically disabled young people using electric powered indoor/outdoor chairs (EPIOCs). Methods: A priori interview questions examined young people’s functioning with EPIOCs, pain and discomfort with ...
De Souza, LH +3 more
core +2 more sources
Cytoskeleton, EarlyView.ABSTRACT Of the three types of cytoskeleton known in animals—actin, microtubules, and intermediate filaments—only actin and microtubules exist in plants. Both play important roles in cellular shaping, organelle movement, organization of the endomembrane system, and cell signaling.
Norman R. Groves +3 more
wiley +1 more source
State-of-the-art Advances in Duchenne Muscular Dystrophy
European Medical Journal, 2017 Duchenne muscular dystrophy (DMD) is a severe and fatal muscle condition affecting young children. Without interventions, affected boys lose the ability to walk independently by the age of 10 and develop progressive cardiac and respiratory failure.
Henriette Van Ruiten +2 more
doaj
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells. [PDF]
, 2013 Duchenne muscular dystrophy is a progressive and incurable neuromuscular disease caused by genetic and biochemical defects of the dystrophin-glycoprotein complex.
Borges, Luciene +11 more
core +2 more sources
Clinical Pharmacology &Therapeutics, EarlyView.Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami +2 more
wiley +1 more source
Increased levels of interleukin-6 exacerbate the dystrophic phenotype in mdx mice [PDF]
, 2015 Duchenne muscular dystrophy (DMD) is characterized by progressive lethal muscle degeneration and chronic inflammatory response. The mdx mouse strain has served as the animal model for human DMD. However, while DMD patients undergo extensive necrosis, the
Berardinelli, Maria Grazia +10 more
core +2 more sources
A Fast Path from Innovation to Safe and Effective Medicine
Clinical Pharmacology &Therapeutics, EarlyView.Consistent progress in medicines development has allowed both for de novo treatment options and for the refinement of existing products that improve effectiveness or reduce harm. Nonetheless, unmet medical needs persist, particularly in rare diseases, pediatrics and underserved populations.
Peter Arlett +11 more
wiley +1 more source