Current Concepts and Emerging Treatments in Neuromuscular Diseases
A special issue of Applied Sciences (ISSN 2076-3417). This special issue belongs to the section "Applied Biosciences and Bioengineering".
Deadline for manuscript submissions: closed (30 September 2021) | Viewed by 389
Special Issue Information
Dear Colleagues,
The field of neuromuscular diseases (NMD) has seen tremendous development in recent years. The treatment landscape has changed to the extent that we need to reconsider our basic premises on best practices and approaches to the treatment of NMD. With new emerging disease-changing therapies, diseases that used to be fatal in the first years of life, now treated early, can become diseases that we can manage, instilling greater hope to patients and their families.
However, in order to do so, we need to improve the early detection of NMDs and increase our understanding of underlying mechanisms. The use of novel biomarkers could also represent an important avenue towards improving treatment. They can improve our understanding of whether patients are responding to treatment in a favorable way and whether other (combinations of) treatments could ensure a better outcome. Precise monitoring of disease progression is also likely to become even more important for detecting changes that require additional management, helping to ensure improvements in the daily functioning of patients and the quality of life of patients and their families.
The main objective of this Special Issue is to give an overview of current best practices and provide suggestions on promising developments in the field.
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Keywords
- neuromuscular diseases
- spinal muscular atrophy
- Duchenne muscular dystrophy
- novel treatment options
- emerging therapies
- newborn screening
- psychological assessment
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