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My WebMD Show Menu My Tools My WebMD Pages My Account Sign Out FacebookTwitterPinterest WebMD Home Health News Email a FriendPrint Article Information and Resources This article is from the WebMD News Archive New Muscular Dystrophy Treatment Offers Hope Study Shows Patients With Duchenne?s Muscular Dystrophy Are Walking Better With PRO051 Treatment By Daniel J. DeNoonWebMD Health News Reviewed by Laura J. Martin, MD March 23, 2011 ? Exciting findings from an early-stage clinical trial offer new hope to patients with Duchenne?s muscular dystrophy, the most common but incurable and devastating form of muscular dystrophy. Patients who received three months of weekly injections with PRO051 had a modest improvement in their ability to walk, reports a research team led by Judith C. van Deutekom, PhD, vice president for discovery at Prosensa Therapeutics, which funded the study. ?The patients are still on the product, and anecdotal evidence suggests they are functioning better in daily life,? van Deutekom tells WebMD. Duchenne?s muscular dystrophy (DMD) is a very common form of the disease, striking about one in every 3,500 boys worldwide. It?s a recessive genetic defect, carried by mothers but almost always striking boys. The defect is in the gene coding for dystrophin, a protein with a major role in muscle function. Normal dystrophin acts like a shock absorber for muscle cells. The defective dystrophin in DMD leads to muscle damage and degeneration, says muscular dystrophy NLM Roger W. Kula, MD, head of the neuromuscular clinic at North Shore-Long Island Jewish Health System. ?Duchenne?s patients have progressive wasting of muscle,? Kula, who was not involved in the PRO051 study, tells WebMD. ?The disease affects walking at age 2 to 5 years, confines patients to wheelchair by age 9 to 12, and results in death from age 20 to 30. It is horrible.? Different patients with DMD have different mutations in the gene that codes for dystrophin. One of the most common mutations ? seen in about 13% of DMD patients ? is at a site called exon 51. PRO051 is a string of nucleic acids called an antisense oligonucleotide. When the body is decoding the dystrophin gene, the drug causes the mRNA that is ?reading? the gene to skip over the mutated exon 51. The result is a dystrophin protein that isn?t exactly normal, but which works well enough. Muscular dystrophy patients who have naturally occurring dystrophin like this have a much milder form of the disease known as Becker?s muscular dystrophy. In their study, van Deutekom and colleagues enrolled 12 DMD patients at an average age of 9 years. In the first phase of the study, they gave increasing doses of the drug to sets of three patients. There were no serious safety issues, and patients given higher doses showed evidence of making functional dystrophin. This led to a second phase in which all 12 patients received weekly abdominal injections of PRO051 at the highest dose tested. ?We noticed a modest improvement, which is quite remarkable for patients with this disease, in the distance they can walk in six minutes,? van Deutekom says. ?The dystrophin probably accumulated over time in their muscles and led to the observed improvement.? 1 | 2 Next Page > Further Reading: Duchenne?s Muscular Dystrophy Advance Stem Cells May Treat Muscular Dystrophy Creatine May Help Muscular Dystrophy Muscular Dystrophy Diagnosis & Treatment Symptoms of Muscular Dystrophy Facioscapulohumeral Muscular Dystrophy New Drug May Treat Muscular Dystrophy See All Muscular Dystrophy Topics Muscular Dystrophy Home Medical Reference Features News Archive Top 12 Topics 1. Depression Signs 2. Teen Acne Tips 3. Psoriasis and Diet 4. Look Younger Secrets 5. Adult Vaccines 6. Psoriasis Skin Care 7. Ulcerative Colitis 8. Toothbrush Truths 9. Birth Control 10. 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RA & Biologics FROM CBS NEWS Just one week of therapy may help stutterers Study shows one week of therapy was able to help stutterers improve speaking ability and changed how their brain sent signals More from CBS News: CDC: 129 new cases of swine flu this week Utah boy?s sinus issues may have been stuck Lego Study: Stressed men prefer heavier women WebMD Video: Now Playing Bug Bites Bug Bites Tired of those annoying bugs and dangerous insects? Get the buzz on how to avoid the painful stings of summer. Fresh Breath Tips Fresh Breath Tips An NLM offers tips on how to keep your breath fresh. Teeth Bleaching Hazard for Kids Teeth Bleaching Hazard for Kids Dentists warn that bleaching teeth before they are fully developed can lead to big problems. Fish Oil Supplement Benefits Fish Oil Supplement Benefits You can get your fill of healthy omega-3 fatty acids by taking a supplement. Grills and Tongue Piercing Grills and Tongue Piercing Some patients find that sporting a mouth full of metal isn?t all it?s cracked up to be. Subscribe to WebMD Newsletters WebMD Daily Women?s Health Men?s Health Weight Loss Wisdom Submit Sign up for more topics! Health Solutions From Our Sponsors Alzheimer?s Progression Child?s Hearing Loss? Birth Control for Moms Diagnosed With Low T? Options for Depression Bipolar Disorder? Severe Hearing Loss? 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