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Readable research linked to original sources
Browse normalized, publication-ready research with direct links to its evidence and source.
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Readable research linked to original sources
Browse normalized, publication-ready research with direct links to its evidence and source.
3 articles
Newest firstDuchenne muscular dystrophy (DMD) is a severe and progressive form of muscular dystrophy caused by mutations in the dystrophin gene. We previously observed that loss of dystrophin in human induced pluripotent stem cell-derived cardiac fibroblasts (hiPSC-cFib) dysregulated the actin network and induced a metabolic remodeling associated with an exacerbated myofibroblast phenotype. The endocannabinoid signaling (ECS)…
Open article record in new tab ↗Duchenne Muscular Dystrophy (DMD) is a very severe X-linked dystrophinopathy. It is due to a mutation in the DMD gene and causes muscular degeneration in conjunction with several secondary co-morbidities, such cardiomyopathy and respiratory failure. DMD is characterized by a chronic inflammatory state, and corticosteroids represent the main therapy for these patients. To contradict drug-related side effects, there…
BACKGROUNDTraditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). OBJECTIVETo identify how value assessment frameworks may need to be adapted to measure the value to society of DMD therapies. METHODSThree groups of stakeholders (…