Neuromuscular Disease Information
Our mission is to accelerate development of effective treatments and to establish best practice in diagnosis and care for neuromuscular patients worldwide. You can learn more about the neuromuscular disease areas that we support at TREAT-NMD below.
We are continuing to update our disease overview pages. If you would like to be involved in this process or have any suggestions for updated content please email info@treat-nmd.com
Muscular Dystrophies
The muscular dystrophies are a group of diseases that cause weakness and degeneration of the skeletal muscles.
Becker muscular dystrophy (BMD)
Congenital muscular dystrophy (CMD)
Duchenne muscular dystrophy (DMD)
Facioscapulohumeral muscular dystrophy (FSHD)
Limb-girdle muscular dystrophies (LGMD)
Myopathies
A myopathy is a disease of muscle in which the muscle fibres do not function properly, resulting in muscular weakness.
Centronuclear & Myotubular myopathies
GNE myopathy/Nonaka myopathy/hereditary inclusion-body myopathy (HIBM)
Motor neuron diseases
In motor neuron disease, nerve cells called motor neurons progressively lose function, causing the muscles they control to become weak and then non-functional.
Neuromuscular junction diseases
Neuromuscular junction disorders result from the destruction, malfunction or absence of one or more key proteins involved in the transmission of signals between muscles and nerves.
Congenital myasthenic syndromes (CMS)
Peripheral nerve diseases
In peripheral nerve diseases, the motor and sensory nerves that connect the brain and spinal cord to the rest of the body are affected, causing impaired sensations, movement or other functions.