Experimental Therapies

I may not be able to save her, but I can give her the best life possible.

The father of a 5-year old with Leigh's Disease, a mitochondrial condition, talks about how his job is to do all he can to help his daughter, including with experimental trials.

When your child is in an experimental clinical trial ….

The mother and father of a 5-year old with Leigh's Disease, a mitochondrial condition, talk about how their daughter's positive response to an experimental study drug means she has more time but they don't know how much or what to expect. "When she gets sick, we don't know if this is going to be the one."

Finding an experimental trial, study drug.

The mother and father of a 5-year old with Leigh's Disease, a mitochondrial condition, talk about they did loads of research online, on their own, including Google Alerts, and learned about an experimental trial. "We had to educate ourselves in order to provide the best for our child."

Sanfilippo/MPS: We hoped she would live to see a cure.

The mom of a child with Sanfilippo describes how they thought about experimental trials -- Our goal was always quality of life, but if this prolonged her life by some years, then that would give more opportunity for a cure to be available. Her decline had been so slow. Eventually we probably realized it wasn't coming in time but there was always that hope.

I may not be able to save her, but I can give her the best life possible.

When your child is in an experimental clinical trial ….

Finding an experimental trial, study drug.

Sanfilippo/MPS: We hoped she would live to see a cure.