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TREAT-NMD

TREAT-NMD

Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world

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Neuromuscular Disease Information

Home / Resources and Support / Neuromuscular Disease Information

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) 

Myotonic dystrophy (DM) 

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. 

Spinal muscular atrophy (SMA) 

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. 

Charcot-Marie-Tooth disease (CMT) 

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  • Resources and Support
  • Disease Information
    • Neuromuscular Disease Information
    • Becker muscular dystrophy
    • Charcot-Marie-Tooth
    • Congenital muscular dystrophy
    • Congenital myasthenic syndromes
    • Duchenne muscular dystrophy
    • Facioscapulohumeral muscular dystrophy
    • GNE myopathy
    • Limb girdle muscular dystrophy
    • Myotonic dystrophy
    • Myotubular and centronuclear myopathies
    • Spinal muscular atrophy
  • Research Overview
    • Research Overview
    • Gene Therapy Research
    • DMD
      • DMD
      • Gene Therapy for DMD
      • Mutation Specific Approaches
      • Cell Therapy
      • Drug Therapy
  • Standards of Care & Family Guides
    • Care Guides
    • CM Care
    • CMD Care
    • DM Care
    • DMD Care
    • FSHD Care
    • SMA Care
  • Standard Operating Procedures (SOPs)
    • SOP Library
    • MDX Mouse (DMD)
    • GRMD Dog (DMD)
    • CMD Mouse (CMD)
    • SMA Mouse (SMA)
    • SOP Archive
      • MDX mouse (DMD) Archive
      • SMA mouse (SMA) Archive
      • Mouse model (DMD) Archive
  • Social and Ethical issues
    • Social and Ethical Issues
    • Concerns Over Stem Cell Therapy for SMA Type 1 Children
    • Early Access to Medicines in Development
    • Ethical Questions

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  • Resources and Support
    • Neuromuscular Disease Information
      • Becker muscular dystrophy
      • Charcot-Marie-Tooth
      • Congenital muscular dystrophy
      • Congenital myasthenic syndromes
      • Duchenne muscular dystrophy
      • Facioscapulohumeral muscular dystrophy
      • GNE myopathy
      • Limb girdle muscular dystrophy
      • Myotonic dystrophy
      • Myotubular and centronuclear myopathies
      • Spinal muscular atrophy
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    • Research Overview
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        • Gene Therapy for DMD
        • Mutation Specific Approaches
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      • CM Care
      • CMD Care
      • DM Care
      • DMD Care
      • FSHD Care
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    • SOP Library
      • MDX Mouse (DMD)
      • GRMD Dog (DMD)
      • CMD Mouse (CMD)
      • SMA Mouse (SMA)
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