“I wouldn’t want anyone to suffer from it, that’s what I would like to see. Early newborn screenings so people can [access] treatment a lot quicker. Definitely having something available would be fantastic so people won’t have to suffer.”

“Involve us. At the very core of decisions, ask us and don’t just sit in an office and make decisions for us without being connected with who you’re making decisions about.”

“Please, please, please help bring Spinraza here. I don’t think anyone can imagine how heartbreaking it is to know there is a potential treatment out there that could slow this thing down, but not be able to access it.”

“These people are all valid human beings, and they need resources. You need to support the families that support them.”

“Firstly, have they ever seen an SMA child? And if not, what’s down on paper is not what’s involved. They really need to see what a debilitating and life-threatening disease it is.”