Meet Regina and Al. Their friendship and respect for one another all started at a fundraiser before a Phillies baseball game in 2019. “I watched Regina give a beautiful speech at the event about her recently diagnosed son, Shane,” recalled Al. “I thought to myself, ‘My friends at Cure SMA need to meet this amazing mom and get her involved.’”
Thanks for posting this. Although this isn’t an ideal solution I do feel like it’s a step in the right direction. In a perfect world everyone who needed it would have access. Despite this program expanding access I think there will still be hurdles and of course 100 treatments isn’t nearly enough. It is better than nothing though.
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.
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