On the cover: Amy Dunaway-Haney of South Padre Island, Texas, was found to have limb-girdle MD at age 8 and began using a wheelchair in her teens. Now married, she teaches high school Spanish and has been nationally honored for her teaching and as the 2003 recipient of the MDA National Personal Achievment Award.Dear Friends
What is Limb-Girdle Muscular Dystrophy?
What are the forms of limb-girdle muscular dystrophy?
How fast does LGMD progress?
What happens along the way?
What's not affected in LGMD?
What tests are used to diagnose LGMD?
Is it important to find out what genetic type of LGMD a person has?
Can special diets help in LGMD?
Are there any special exercises that can help?
Known Forms of Limb-Girdle Muscular Dystrophy
Does it Run in the Family?
MDA's Search for Treatments and Cures
MDA is Here to Help You |
MDA's Search for Treatment and Cures
Limb-girdle muscular dystrophy is a fertile area for MDA-sponsored research.
In the recessive forms of LGMD, it may be possible to some day insert a new gene to compensate for one that isn’t working properly. Known as gene therapy, this type of intervention is now being tested in people with another form of muscular dystrophy. A clinical trial to test the safety of transferring functional alpha-sarcoglycan genes to a muscle in people with LGMD due to mutations in this gene is expected to begin in 2007 or 2008.
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In the dominant forms of LGMD, it may be possible to block a toxic gene. This kind of strategy will be tested in 2007 in another disease.
Some genetic mutations, known as “premature stop codons,” cause cells to stop reading genetic instructions too soon before a fully functional protein has been synthesized. A drug that causes cells to “read through” these stop codons is being tested in another form of muscular dystrophy and may have application to some LGMD patients.
Other genetic mutations add or remove DNA and change the way cells interpret the information in a gene. In another form of muscular dystrophy, clinical trials of compounds that coax cells to snip out these error-containing DNA regions and read the rest of the gene are under way as of 2007. These too may eventually have implications for LGMD treatment.
Another avenue of investigation is blocking a natural protein called myostatin, which puts a brake on muscle growth. A myostatin-blocking drug is being tested in people with LGMD and other forms of MD.
Still another idea is to use stem cells, primitive cells found in the bone marrow and muscles, to help ailing muscles regain strength. These cells could, in the future, be used to carry new genes to the muscle cells, or just to replenish their capacity to repair themselves even without a complete set of working genes.
MDA-supported researchers worldwide continue to pursue every avenue leading to potential treatments.
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