Sheryl Marrazzo, a Duchenne caregiver advocate, discusses weighing the benefits and risk factors of starting a child with MD on a new medication, and advises caregivers on what questions to ask their providers.
Transcript
So I think you need to collectively weigh the risk-benefit profile of any medication. And I think when you approach your doctor, I don’t think you’re completely decided with what you want to do. It’s a team conversation.
I’m very lucky where I work with scientists who have been in the rare disease space for 20, 30 years. So I actually can just phone a scientist. Not everyone has that luxury, but I do believe, if you reach out to your friends and family on social media, ask them questions, but also realize that they may have had a different experience than you will have.
You look at the risk-benefit. Many of the treatments that are approved are not—my son’s not amenable to. But when you look at what’s available in clinical trials, there’s a lot to decide. And there’s that risk-benefit profile. “Is it safe? Is it, you know, effective? And what will happen, side effect-wise, or what have you, right? And is that risk-benefit worth it?”
And then when your child gets older, they also need to come into that. When you look at treatments, “How often do you need to get the treatment? Is it every week? Is it every month? Is it twice a year? You know, what is that burden for the family? You know, is it worth it?”
So I think you kind of need to do that work first before you go to your doctor to ask the questions because you need to know: “Can my family do this? Is there too much of a burden? Is the risk-benefit profile not something that we’re willing to take?”
But then go speak to your doctor and sort of get their take on it. And you’d be surprised at many different physicians in the same field might have a different opinion.
