[adela-mohanu]

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 therapy, absolutely nothing to blame here. But should someone without access to treatment die because he/she didn’t have so much luck at a draw? That’s a question the company should answer, not the parents.

[adela-mohanu]

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 with a 15mo son who is sma1 and I think that the next weeks will also give a trend for Europe. While I hope it will be soon approved, in Europe we have different kind of insurance policies and basically the governements have to be willing to sustain the costs. I fear that they will look at any rejection in US and use it as an argument.

Joyfull days to you all!