Massachusetts Personal Injury Library
Hope for Muscular Dystrophy Patients
A recent article in The Boston Globe says that a Brown University research scientist, a dedicated medical doctor, and a mother who wouldn’t give up hope, have collaborated to create lab-grown muscles to test treatments for muscular dystrophy (DMD).
A mother’s search for answers
Christine McSherry of Pembroke, whose son, Jett, 13, has the disease, was told the genetic disease would kill her son by the time he was 15. Doctors told her when they diagnosed her son that there was absolutely nothing she could do, the disease is progressive and there is no cure. She found that answer unacceptable, and instead formed a nonprofit organization called the Jett Foundation to help fund DMD research and began to find doctors who were aggressively treating the symptoms.
The mother describes how she met Dr. Brian Tseng in 2006, a molecular biologist and doctor who specialized in DMD. Dr. Tseng was asked to join Harvard Medical School’s faculty and start a DMD clinic and lab at MassGeneral Hospital for Children. McSherry used her foundation’s funds to help open the clinic.
Looking for alternative treatments
Dr. Tseng said in the article that DMD is a difficult disease to study because of the lack of good clinical models. The disease affects mice differently than it does humans, and dogs are too costly to work with, said the Globe. The doctor is always looking for alternative treatments.
Dr. Tseng met Brown Professor Herman Vandenburgh who is experimenting with the growth of tiny muscles for testing alternative treatments. These experiments will allow Dr. Vandenburgh to tell exactly how strong the muscle is and how effective various treatments are, according to the article.
Hope for muscular dystrophy treatments
Dr. Tseng said human trials will be able to begin by the end of the year on effective treatments. And McSherry’s foundation is raising money through various fundraising activities to cover the $150,000 for the screening process and $600,000 for human trials, said the article.
McSherry said the collaboration with Vandenburgh and Tseng has allowed doctors to keep children with DMD healthier for longer, and they will be able to benefit more from eventual gene-therapy or stem-cell treatments. She said she now is planning her son’s college education and career because she is convinced he has a future, said the article.
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