I have two sons with Duchenne. They are both students at MSU. My oldest son will get his master's in Molecular Cell Biology in the spring. They both struggle every day just to live. They are unable to care for themselves, so they must depend on someone to care for most of their needs. They are both extremely smart and have worked really hard to stay alive. The new medicine could enable them to live by themselves and have lives of independence. Needing help means they depend on help to get in and out of bed, on and off the toilet, getting food, etc... They have struggled their entire lives.
Please help these brilliant young men! They know if they don't get help within the next few years, their future is death. They could help our society with their brains.