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The muscular dystrophies are a group of genetic diseases that affect skeletal muscles and often also heart muscle. The symptoms include muscle weakness and progressive muscle wasting.

The major forms of muscular dystrophy are:

Myotonic
Duchenne
Becker
Limb-girdle
Facioscapulo-humeral
Congenital
Oculo-pharangeal
Distal
Emery-Dreifuss

Duchenne muscular dystrophy (DMD) is the most common and a very severe form of the disease. It is caused by a genetic fault which prevents the production of a protein called dystrophin. Without dystrophin, muscles are fragile and are easily damaged. Over time so much damage builds up that the body can’t repair it and muscles waste away, causing progressive disability in patients.

In patients with a genetic disease like muscular dystrophy, stem cell therapy is a very attractive option because injection with a specific type of “stem cell” that can make muscle and express the missing protein has the potential to repair abnormal muscle function.

How could stem cells help?

Producing healthy muscle fibres with the help of:

Myoblasts - a type of cell formed from satellite cells
Mesoangioblasts (MABs) - a type of stem cell or 'progenitor' cell.
Induced pluripotent stem cells (iPS cells) - made from skin cells in the lab and are pluripotent

Reducing inflammation with the help of: