Thanks for your inputs.
My opinion is that the approach is unethical. There are children who don’t have access to any kind of treatment or that have severe side effects frum current treatments. They just might lose the lottery to someone who is already thriving with other available meds. Of course every parent will want to have access to gene… Read more

So Novartis made it clear: there will be a lottery for the 100 doses it offers out of USA. What’s your opinion about it?

https://investors.avexis.com/news-releases/news-release-details/avexis-community-statement-global-managed-access-program-for-avxs-101

Hello everybody,

I was wondering if anyone started the process of receiving Zolgensma. What are the steps? Are the doctors willing to prescribe it? Do the companies accept the prohibitive price?

It would be nice to find out about the first patient who received it not in a trial, but through a regular insurance.

We are an Italy-based family… Read more