Spinal Muscular Atrophy in Twins

Our twin daughters were born apparently healthy but when they turned 10month old, they were diagnosed positive for SMA (spinal muscular atrophy) type 1. It's a rare and agressive type of genetic disease. There is a new and revolutionary drug that was approved by FDA last year, but it has to be taken before their second aniversary in order to be able to correct their genetic defect. This medicine is called Zolgensma (Novartis/Avexix) and unfortunetly it is the most expensive medicine in the world, costing 2.1million dolars. In our case having twins, 4.2millions.
Our daughters are already 1year and 6month old, having only a few months left to recieve this treatment.