The availability of animal models that mimic human diseases has greatly increased our understanding of neuromuscular rare disease pathology.
Neuromuscular diseases are a heterogeneous group of rare inherited disorders that impair peripheral nerve and/or muscle function and eventually lead to muscle weakness, wasting, and other complications. Neuromuscular diseases include a wide range of rare disorders, including amyotrophic lateral sclerosis (ALS), Huntington’s disease, neuromuscular junction disorders myasthenia gravis, and more. With limited treatment options available, there is a growing need to design effective preclinical studies to achieve greater success in clinical trials of new therapies.
Recent advances in genetics and molecular biology have led to the development of animal models that mimic the symptoms of these diseases. These models provide valuable insight into their underlying mechanisms. Although animal models do not fully recapitulate human pathology and there are differences in disease severity between them, they are essential for assessing drug safety and efficacy and for helping prioritize compounds for further clinical development.
Animal Models of ALS Disease
Animal Models of Huntington’s Disease
Animal Models of Neuromuscular Junction Disorders
Animal Models of Sarcoglycanopathy
Animal Models of Duchenne Muscular Dystrophy
Animal Models of Calpainopathy