Bobby was diagnosed with suspected dense deposit disease (DDD) at 11-years old. DDD is a rare genetic disease of the kidneys. It affects about 2-3 people per million and symptoms usually begin between the ages of 5 and 15. 50% of children with DDD will suffer kidney failure within 10 years.
DDD is exceptionally difficult to diagnose, and it is often mistaken for several other similarly rare kidney diseases in children. There are only two experts in the world in DDD: one is a physician in France, and the other is Dr. Richard Smith of the University of Iowa. When Dr. Smith heard that he had a case, he implored us to send the case to his team immediately so that they could apply their full breadth of experience. His team included another partner, Dr. Carla Nester.
There is not yet a cure for DDD, but there is a standard treatment. The standard treatment is a combination of an immunosuppressant and a steroid. The limited data on this drug cocktail suggests that up to 30% of patients respond to the treatment.
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Bobby’s treating physicians had initially recommended that he remain on the standard treatment indefinitely. But Dr. Smith and Dr. Nester believe that every child deserves at least one chance to be off medications. They recommended keeping Bobby on the drug cocktail for two years while reducing the steroid dose by 5 fold. They would then test to see whether he had responded to the drug cocktail and could stop taking medication safely.
Our medical experts also noted that in the event Bobby’s symptoms worsened, there is a new biologic therapy that may help save his life. This therapy is not approved for use in DDD or similar diseases. MORE Health regulary reviews clinincal trials under way in the United States and can facilitate requests for joining those trials.
“This patient illustrates MORE Health’s ability to recruit the best consultants in the US for rare diseases.”
Today, Bobby’s condition is well managed, and he is enjoying a higher quality of life with a lower steroid dose.
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