Question: Can you treat FOP disease?

Richard Prince answered on 18 Jun 2015:

Stone man disease….it’s so rare that there have not been huge efforts to treat it. However, the mutation that causes the disease has been identified so you can make animal models (transgenic mice) with the disease. Two types of treatment might be possible. One would be to try to find drugs to stop the mutated protein from working. The second would be to use small bits of RNA that will bind to the mRNA for the mutated protein and stop it being translated into protein. This is known as RNA interference (RNAi). RNAi has potential in all kinds of diseases and is actually one of the ways in which the body downregulates genes naturally.
Another more immediate way to deal with FOP is to offer the parents genetic counseling and perhaps to do IVF, screening the embryos before implantation. It’s an autosomal dominant disorder: assuming the affected parent is heterozygous, and the other parent is not affected, the children would have a 50:50 chance of being affected. However, most cases are caused by spontaneous mutations in the gametes of normal individuals, so this would not make a huge difference to the number of cases.
Best wishes
Richard