volsain

So...uh...did you bone any of the princesses?

May I ask what process was used to determine that you have an unspecified form of MD? I actually research a type of MD called Facioscapulohumeral Muscular Dystrophy, it's the third most common type of MD. Our group is making process in a drug that may be the magic bullet that most are hoping for. It's shown promise in Duchenne (experimental, our center developed it and is going for clinical studies soon) and from what we have noticed from our experiments, in FSHD as well. I am not sure whether this will be something that can benefit you but if I had to put my money on one drug that can, it's this one.

I apologize for the late reply. The drug is a natural compound and we are about to start clinical trials in children. We are hoping to get it fast tracked but only the results will determine that. Hopefully, in about 5 years we may be able to get it past all the hurdles.

Since we (not me but the lab in our center) are going to be testing in children first it will have to go through that trial first. I am sure it will work in adults as well. The drug is actually not very well known. So little in fact that only our center knows about it for the most part.

I'm sorry for the delayed response. I was in bed and fell asleep. I can't disclose the name of the drug yet, but it is a natural compound. This was found way before I got here by one of the founders of the Dystrophyn gene. Because of the way the drugs have to go through validations, toxicology analysis, animal studies, etc. it's very expensive. If you go through clinical trials involving adults there are huge fees slapped on you. That's the reason that the FDA can operate during the government shutdown, their source of funding are these fees (at least to my knowledge). So first step is to test in children aged 5-7 years. Because their organs are still developing the side effects would show. We don't anticipate very many sude effects, if at all. However, things can come up. I'm thinking it will take about 6 more years. A lot of it is also due to funding. Big Pharmas want some results before investing, venture capitalists can fund now but we can't trust their intentions for future direction. They may be focused on the $ rather than what's important.

For FSHD we also have another natural compound that has shown suppression of the protein which causes FSHD. Since you can find it in food naturally, there won't be as much of an issue with it if we can produce more data. As selfish as this may seem, we are hoping to patent the methodology. This way, when the methodology is used to treat the disease, we can either get some money in the future to help us fund the experiments in our lab or we can make it available free to public so that big Pharmas can't make much money off of it, if at all.