There was an article last November saying Zolgensma could be helpful for adults. Will they be doing a clinical trial on that soon? I have not seen anything further on it.

I am 40+ years old, have held jobs, am reasonably intelligent, but I still look like a teenager aside from a few grey hairs. My dad is 83, a little hard of hearing. My attendant, the only reliable attendant I can find in the entire state, is nearly the same age, is physically in much better shape, but is even harder of hearing. So I was dealing with my father who gets very grouchie when he hasnt eaten trying to help him with cooking which he doesnt know how to do and caregiver of course walks… read more

Growing up, at what age did you understand or had been told you have not something wrong with you but that you have special muscles unlike the other kids? (Thats what we call connors condition, to help his older 8 yr old brother understand) connor is type 1 and is currently 7 months old. Recvd zolgemsa on halloween. Didnt finish our last loading dose of spinraza. If you currently do maintaince dosing of spinraza has it been successful for you? Pro? Cons? Effectiveness still? At what age did you… read more

Hi, does someone in the team has an idea about ataxia telangiectasia. It’s a rare neuro degenerative disease and not curable for the moment. I would like to know if there are any studies or tests in course. I’m personally following a young bit from Vietnam since 7 years. He 14 now. When I first saw him he was running and enjoying. Today he’s bed ridden and needs assistance for everything. Thank you

How do you earn money without loosing critical disability benefits and Medicare ? So, in many ways, I am very lucky. When I started needing attendant care, I did the math and there was no way I could earn enough money, keep working, and pay for attendant care. The programs in my state that cover attendant care very strictly require an individual have less than $2,000 in assets total (including such things as gold teeth, burial plots, etc) and not be employed. If not for my father's support… read more

I have been suffering from a lack of oxygen in the blood for two months, and I started using supplementary oxygen and I did not feel better. For those who went through this experience, what did you do during the lack of oxygen?

Hi guys , I am 22 years old.I live in the countryside of Malaysia.I'm here today to ask a small question. but my english level quite bad, hope you all who read this post can take some patient to understand my voice.

Since I didn't have a university, I only finished my second year of high school.I have graduated for almost four years，but I never know how my future will happen .To be honest, I always thought I was an ordinary person, because I went to an ordinary high school, and I only realized… read more