Muscular dystrophy is the most common inherited neuromuscular disorder among children affecting 10,000-12,000 children in the U.S., mostly males. Most children end up in wheelchairs prior to their teenage years due to muscle destruction. The next phase of the disease is the muscles in the heart and lungs also giving out and no longer working properly, leading to people dying before their 40s. There is an experimental gene therapy for Duchenne muscular dystrophy now, which works by infusing trillions of harmless viruses that have been genetically modified to a gene in patients' muscles. The gene would then produce a miniature version of the dystrophin protein (which is the protein missing or the limit in people with muscular dystrophy), and the hope is that this micro dosage of the protein would help to slow the progression of the disease. The treatment is in the process of getting FDA approval, and the other concern is cost since this type of gene therapy would cost around $3 million to treat each patient, which may not always be covered by insurance.
This gene therapy could be a great idea if it is able to successfully slow the progression. Many children are wheelchair-bound at such a young age from this disease and people are unable to live the majority of their lives due to a shorter lifespan, so having this gene therapy as a possible progression slower would be a huge advancement in the treatment of patients with muscular dystrophy. The only negative side of this treatment would be the price, but the price is a small price to pay when you save people's quality and length of life.
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