First identified in the 1860’s by neurologist, Guillaume Duchenne, Duchene muscular dystrophy (DMD) is the most common, most lethal member of the muscular dystrophy family, affecting approximately 1 in every 2,400 boys.
What is DMD?
In DMD, abnormal genes (mutations) interfere with the production of proteins needed to form healthy muscle. This causes weakness and loss of muscle mass. DMD also affects breathing and heart functions.
Some who have it will eventually lose the ability to walk. Some will have trouble breathing and/or swallowing.
Because there is no cure, it remains an anguishing challenge for the patients, as well as their families dealing with this debilitating disease.
Much effort has been put into the advancement of research into muscular dystrophy so that now, medications and therapy can help manage some of the symptoms and slow the course of the disease.
Researchers well-established in the treatment of Duchenne muscular dystrophy have new understanding of the benefits of steroids in the treatment of DMD with clearer guidance on their use. Corticosteroid treatment helps to improve muscle strength, maintain breathing functions, stabilize ambulation, prevent spinal deterioration (scoliosis) and delay onset of heart disease.
“Eureka Alert!” has proclaimed that a gene-editing technique successfully stopped the progression of Duchenne muscular dystrophy in a mouse model. Gene editing or gene splicing is called CRISPR.
The researchers used CRISPR technology to delete exon 23 from the Duchenne gene on the X chromosome. Exon 23 is the site of the fault that makes DMD patients unable to produce the muscle protein called dystrophin.
By splicing this exon out, the researchers demonstrated an increase in the production of dystrophin. This increase was significant enough to lead to an improvement in muscle strength.
At the moment, only mice have experienced the advantages of this technology. Could it be that exon skipping therapy is the wave of the future for DMD sufferers?
Enter the Nootropics
On the front lines of neuro-medicine are the nootropics. One in particular has contributed to the treatment of Alzheimer’s and been proven successful with heart and liver disease. Recently, researchers have enlisted it in the war on DMD.
Idebenone/Lucebanol is a nootropic product and derivative of Co-enzyme Q10, initially developed for the treatment of Alzheimer’s. It is also used for liver disease, and heart disease as well as Leber’s disease (an eye condition), mitochondrial encphalyomyopathies (nerve and muscle disorders) and Friedreich’s ataxia.
Because Idebenone protects a wide variety of cells from oxidative damage, it has found a purpose in those suffering from multiple sclerosis.
Researchers at Newcastle have been investigating Idebenone’s vision-preserving effect in the mitochondrial disease called Leber’s hereditary optic neuropathy (LHON). Idebenone is thought to improve the activity of mitochondria, the energy-producing component of all cells.
Idebenone has also been investigated in other neurological disorders such as Friedreich’s ataxia.
There have been similar benefits in respiratory function in DMD patients using Idebenone.
Advantages of Idebenone
Idebenone/Lucebanol with its ability to increase the activity of the mitochondria is an important ally for all those who suffer from DMD.