Neuromuscular Disorders Drug Discovery Service
Neuromuscular disorders affect the quality of life of many people in the United States and cause serious social problems. Creative Biolabs is a leading services provider in the field of neuroscience. We are committed to solving all problems for our customers.
Introduction of Neuromuscular Disorders
The peripheral nervous system is composed of nerves that branch out from the brain and spinal cord. Neuromuscular disorders refer to a wide range of diseases that affect the peripheral nervous system. When the nerve cells responsible for sending and receiving electrical messages to the body become unhealthy, the communication between the nervous system and the muscles would be affected and eventually lead to muscle atrophy. Symptoms of the neuromuscular disease appear at various stages of life, including infancy, childhood, and even adulthood. Progressive muscle weakness and fatigue are the main symptoms of neuromuscular disorders. Currently, there is no cure for most neuromuscular diseases, but reasonable treatments may relieve disease symptoms, improve quality of life, and prolong life.
Types of Neuromuscular Disorders
Charcot Marie Tooth (CMT) disease, also known as hereditary motor and sensory neuropathy (HMSN) or peroneal muscular atrophy (PMA), is a group of inherited disorders that damage peripheral nerves. CMT can be divided into three forms, including autosomal dominant demyelinating CMT called CMT1, autosomal recessive CMT4, as well as a rare recessive form with severe penetrance that appears in early childhood, which is called CMT3.
Duchenne muscular dystrophy (DMD) is a severe progressive muscle-wasting disease that affects boys and is much rarer in girls. Muscle weakness usually begins around age four and worsens rapidly. Till now, there is no known cure, while gene therapy as a potential treatment is in the early stages of research.
Familial amyloidosis polyneuropathy (FAP), also known as familial amyloidotic neuropathies, neuropathic heredofamilial amyloidosis, or familial amyloid polyneuropathy refers to a rare group of autosomal dominant diseases that the autonomic nervous system and/or other nerves are damaged by protein aggregation and/or amyloid fibril formation.
Mucopolysaccharidoses (MPS) are a group of inherited lysosomal storage disorders. Defects in the activity of lysosomal enzymes lead to incomplete degradation processes and cause abnormal accumulation of heparan sulfate, dermatan sulfate, and keratan sulfate that interfere with cellular function.
Muscular dystrophies (MD) are a group of progressive genetic disorders that cause muscle weakness and even disability. Some types of MD can eventually be life-threatening by affecting the heart or the muscles used for breathing. There is no cure for MD, but various treatment options can help relieve symptoms.
Myasthenia gravis (MG) is a rare long-term neuromuscular disease that leads to muscle weakness. The disease can affect most parts of the body, including the muscles that control the eyes, facial expressions, chewing, swallowing, and speaking. It affects people of any age.
Myotonic dystrophy (DM) is a type of muscular dystrophy that leads to progressive muscle loss and weakness. In DM, muscles often fail to relax after contracting. Other possible symptoms include cataracts, intellectual disability, and heart conduction problems.
Pompe disease is a rare genetic disorder in which the body can't produce proteins to break down a complex sugar. The disease primarily affects the liver, heart, and muscles, and eventually leads to muscle weakness and difficulty breathing.
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in progressive muscle wasting and movement problems. SMA is due to a mutation in the SMN1 gene.
Common Symptoms of Neuromuscular Disorders
- Muscle weakness
- Movement problems
- Balance problems
- Swallowing troubles
- Breathing troubles
- Tingling or pain
Neuromuscular Disorders Drug Discovery Service
Equipped with world-leading platforms and extensive experience, Creative Biolabs provides one-stop drug discovery service for our clients, from the basic target identification to the final IND-enabling, we can meet all the needs of customers.
We are specialized in the in vitro model development, such as iPSC-derived neuromuscular disorders. We also offer several predictive models for preclinical in vivo evaluation of treatments for neuromuscular disorders. We are dedicated to providing the best neuromuscular disorders drug discovery service to promote your project.
Creative Biolabs is a leading service provider that focuses on neuroscience research. We can assist you in designing the best research outline customized to meet the requirements of your programs. If you are interested in our services and products, please do not hesitate to contact us for more details.