I have an appointment with my neurologist on Tuesday to talk to him about trying to get on the “Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)”. This program was announced early in February and the goal seems to get people (who need it sooner rather than later) access to Risdiplam before FDA approval. It is technically called a study, but the criteria for participation seems pretty broad and it appeared like the company is trying to get more evidence to present to insurance companies if/when the FDA finally does approve it. It is open for SMA Type 1 & 2 who are not currently able to receive other treatment for whatever reason, and who (in the opinion of the prescribing neurologist) would benefit by receiving the medication quicker.
My neurologist told me that there is a ton of paperwork for him to submit to allow his facility to participate in this program, but he assured me that he is going to try to get me signed up with it. I considered Spinraza about a year ago, and almost went through with it, but my breathing issues were concerning to my neurologist and I got the feeling that he wasn’t completely comfortable with me going through it. At the time I was finally scheduled to start the injections, the hospital where the injections were to take place had a breathing scare with another individual with SMA and they postponed all future injections until they could retrain all of their staff on how to operate various breathing machines someone may arrive to the appointment with. By the time they wanted to get me back in, I started hearing about Risdiplam and I thought I might as well wait since I was super nervous about the injections anyway. Hopefully, I made the right choice, but now with the coronavirus potentially being a stumbling block to even go to my appointment safely on Tuesday, I’m not so sure.
Anyway, for anyone else who is interested in asking their neurologist to see if they can get early access as well, refer to the following website: https://clinicaltrials.gov/ct2/show/NCT04256265