Introduction/New to group and wanted to contribute

[Guest guest]

Hello everyone please allow me to introduce myself.

My name is Jay Hartz I personally do not have CMT but my wife's aunt does. She

has been diagnosed with Type I and has had multiple surgeries, and has

been in AFOs for as long as I have known her, (6 years). So this has become one

of my goals to learn more about CMT and educate myself about CMT. From

everything I have learned on your site and on the web this disease could affect

my children or there family. So it has become important to me personally.

I have done some research and would like to contribute some

information.

Sincerely,

Jay Hartz

Here's some scientific information,

Two successful double-blind trials with coenzyme Q10 (vitamin Q10)

on muscular dystrophies and neurogenic atrophies.

Folkers K, Simonsen R

Institute for Biomedical Research, University of Texas at Austin

78705, USA.

Coenzyme Q10 (vitamin Q10) is biosynthesized in the human body and

is functional in bioenergetics, anti-oxidation reactions, and in

growth control, etc. It is indispensable to health and survival. The

first double-blind trial was with twelve patients, ranging from 7-69

years of age, having diseases including the Duchenne, Becker, and

the limb-girdle dystrophies, myotonic dystrophy. Charcot-Marie-Tooth

disease, and the Welander disease. The control coenzyme Q10 (CoQ10)

blood level was low and ranged from 0.5-0.84 microgram/ml. They were

treated for three months with 100 mg daily of CoQ10 and a matching

placebo. The second double-blind trial was similar with fifteen

patients having the same categories of disease. Since cardiac

disease is established to be associated with these muscle diseases,

cardiac function was blindly monitored, and not one mistake was made

in assigning CoQ10 and placebo to the patients in both trials.

Definitely improved physical performance was recorded. In

retrospect, a dosage of 100 mg was too low although effective and

safe. Patients suffering from these muscle

dystrophies and the like, should be treated with vitamin Q10

indefinitely.

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Proc Natl Acad Sci U S A 1985 Jul;82(13):4513-6

Biochemical rationale and the cardiac response of patients with

muscle disease to therapy with coenzyme Q10.

Folkers K, Wolaniuk J, Simonsen R, Morishita M, Vadhanavikit S

Cardiac disease is commonly associated with virtually every form of

muscular dystrophy and myopathy. A double-blind and open crossover

trial on the oral administration of coenzyme Q10 (CoQ10) to 12

patients with progressive muscular dystrophies and neurogenic

atrophies was conducted. These diseases included the Duchenne,

Becker, and limb-girdle dystrophies, myotonic dystrophy, Charcot-

Marie-Tooth disease, and Welander disease. The impaired cardiac

function was noninvasively and extensively monitored by impedance

cardiography. Solely by significant change or no change in stroke

volume and cardiac output, all 8 patients on blind CoQ10 and all 4

on blind placebo were correctly assigned (P less than 0.003). After

the limited 3-month trial, improved physical well-being was observed

for 4/8 treated patients and for 0/4 placebo

patients; of the latter, 3/4 improved on CoQ10; 2/8 patients

resigned before crossover; 5/6 on CoQ10 in crossover maintained

improved cardiac function; 1/6 crossed over from CoQ10 to placebo

relapsed. The rationale of this trial was based on known

mitochondrial myopathies, which involve respiratory enzymes, the

known presence of CoQ10 in respiration, and prior clinical data on

CoQ10 and dystrophy. These results indicate that the impaired

myocardial function of such patients with muscular disease may have

some association with impaired function of skeletal muscle, both of

which may be improved by CoQ10 therapy. The cardiac improvement was

definitely positive. The improvement in well-being was subjective,

but probably real.

Likely, CoQ10 does not alter genetic defects but can benefit the

sequelae of mitochondrial impairment from such defects. CoQ10 is the

only known substance that offers a safe and improved quality of life

for such patients having muscle disease, and it is based on

intrinsic bioenergetics.

Muscular Dystrophy: Coenzyme Q:The Ubiquitous Quinone: Part II

This article first appeared in the October, 1993 issues of VRP's

newsletter by A.S. Gissen

CoQ and Muscular Dystrophies

At present, several hypothesis suggest that muscular dystrophy

results from some type of metabolic deficiency. This defect is

manifested by an abnormality in muscle structure that gives rise to

the death of the muscle fibers and to the abnormal muscle

regeneration that is a characteristic of muscular dystrophy. As

early as 1966 it was shown that in mice with genetic muscular

dystrophy, CoQ administration would produce improvement.(28) An

interesting observation in humans was that virtually every form of

muscular dystrophy is associated with cardiac disease.(29) This,

coupled with early success using CoQ in animal models of muscular

dystrophy, led to experimentation with CoQ10 administration in human

forms of muscular dystrophy and neurogenic atrophies.

Muscular dystrophy is not a single disease, but rather, a group of

closely related syndromes. The use of CoQ10 supplementation has been

tried in a large number of these syndromes including the Duchenne,

Becker, and limb-girdle dystrophies, myotonic dystrophy, Charcot-

Marie Tooth disease, and Welander disease.(29) In these patients,

improvements in physical well-being was commonly observed.

Additionally, a direct relationship between muscle and cardiac

impairment was found, as both of these improved on CoQ10 therapy. A

later study, that also included some additional forms of muscular

dystrophies (fascioscapulohumeral muscular diseases and hypotonia

congenitale), also showed improvements in cardiac function for

almost all patients, as well as improved physical performance and

quality of life for many patients.(30) The authors

concluded that therapy with CoQ10 is without any side effect, and

may be given for the lifetime of the patients with muscular

dystrophy. They also noted that there is presently no therapy for

such patients which provides the improvement in quality of life as

does CoQ10.