what is the cure for muscular dystrophy?

Question

I am told that this one has no cure whatsoever. is it true? is there any organization who can help dystophy patients.

Answer 1

Ok how about an actual answer. Is there a clinical treatment to MD? Yes, but it is hardly helpful in preventing the deterioration of muscles. GENE THERAPY, not just stem cells, seems to be the most promising approach to treating MD patients. It has been an intense subject of research for the past 10 years and researchers have come along way.

Basically, the current problem is that the dystrophin gene is one of the longest genes (approx. 9.4 Mb) so introduction of the gene is almost impossible using regular viral vectors (Non-harmful viruses that contain a target gene which the virus delivers to a target cell). Luckily, they have been able to shorten the gene to roughly 14 kb which can fit in viral vectors and still be able to produce viable dystrophin. These types of developments have led researchers to be able to prevent MD in mice limb muscles. However, mice muscles deteriorate at a much slower rate than do humans so the exact process used for mice is not the same for humans. Let us also not forget the multitude of different muscle types in humans, whose body mass is 40% muscle. The treatments that have been developed for the limbs may not work for smooth and cardiac muscles. Therefore, the possibility of using a complicated yet intricate systemic delivery system is currently being investigated. On the flip side, This is promising because the existance of a cure in mice greatly increases the possibility of finding a human analog for gene therapy.

I just did a research presentation on gene therapy for DMD and it's very impressive how far this area of research has developed. I wouldn't be surprised if a human analog is found and human clinical trials begin within the next 10 years.

Answer 2

So far there is no cure- but they have found the enzyme that breaks down the muscle=here is a bunch of info- and web sites--Diane

Facioscapulohumeral Muscular Dystrophy (FSHD) Society
3 Westwood Road
Lexington, MA 02420
[e-mail]
http://www.fshsociety.org
Tel: [phone]
Fax: [phone]

Muscular Dystrophy Association
3300 East Sunrise Drive
Tucson, AZ 85718-3208
[e-mail]
http://www.mda.org
Tel: [phone]
Fax: [phone]


Muscular Dystrophy Family Foundation
3951 N. Meridian Street
Suite 100
Indianapolis, IN 46208-4062
[e-mail]
http://www.mdff.org
Tel: [phone]
Fax: [phone]

Parent Project Muscular Dystrophy (PPMD)
1012 North University Blvd.
Middletown, OH 45042
[e-mail]
http://www.parentprojectmd.org
Tel: [phone]-714-KIDS (5437)
Fax: [phone]


International Myotonic Dystrophy Organization
P.O. Box 1121
Sunland, CA 91041-1121
[e-mail]
http://www.myotonicdystrophy.org
Tel: [phone]

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institutes of Health, DHHS
31 Center Dr., Rm. 4C02 MSC 2350
Bethesda, MD 20892-2350
[e-mail]
http://www.niams.nih.gov
Tel: [phone]-NIAMS (226-4267)


National Institute of Child Health and Human Development (NICHD)
National Institutes of Health, DHHS
31 Center Drive, Rm. 2A32 MSC 2425
Bethesda, MD 20892-2425
http://www.nichd.nih.gov
Tel: [phone]
Fax: [phone]

Centers for Disease Control and Prevention (CDCP)
U.S. Department of Health and Human Services
1600 Clifton Road, N.E.
Atlanta, GA 30333
[e-mail]
http://www.cdc.gov
Tel: [phone]-3543



Related NINDS Publications and Information

Workshop on Theraputic Approaches for Duchenne Muscular Dystrophy
Summary of a May 2000 NINDS workshop on Duchenne muscular dystrophy.
Conference on the Cause and Treatment of Facioscapulohumeral Muscular Dystrophy
Summary of a conference, "The Cause and Treatment of Facioscapulohumeral Muscular Dystrophy," held May 8-9, 2000.
Testimony on Muscular Dystrophy, February 27, 2001
Congressional testimony on muscular dystrophy by Dr. Audrey Penn, Acting Director, NINDS, February 2001.



Prepared by:
Office of Communications and Public Liaison
National Institute of Neurological Disorders and Stroke
National Institutes of Health
Bethesda, MD 20892




NINDS health-related material is provided for information purposes only and does not necessarily represent endorsement by or an official position of the National Institute of Neurological Disorders and Stroke or any other Federal agency. Advice on the treatment or care of an individual patient should be obtained through consultation with a physician who has examined that patient or is familiar with that patient's medical history.

All NINDS-prepared information is in the public domain and may be freely copied. Credit to the NINDS or the NIH is appreciated.

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Last updated October 23, 2006

Answer 3

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Answer 4

There is no cure.
There are some treatments, but there is no cure.

The only hope probably lies in stem cell research.

Answer 5

No cure for it yet, but they are working on it.

http://www.mdff.org/


http://www.mdausa.org/