Top 9 search results for "Zolgensma" in Q&A. To see all results and access other features, sign up for free.

I was wondering is there anyone here that knows someone who or have had zolgensma gene therapy?

Hi everyone. I was wondering if sma adults should start a petition to FDA to make Zolgensma available for adults. The reasons they say we cannot do it: 1) insurance - as adults, most of us have jobs so we have some type of insurance coverage, and the fact the drug is given to kids if insurance does not cover by Novartis anyways is reason why, 2) cost - drug is 2.1 million but most of us on Spinraza or Evrysdi know these drugs cost 400000 a year, so it pays for itself after 10 years, 3)… read more

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.e Spinraza, Evrysdi, Zolgensma

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