Hi, I'm Dr. Moody, a neurologist in Texas who treats SMA. My practice includes children, teenagers, and older individuals living with SMA. Members of the SMA community ask great questions, and today, I'll be sharing my perspective and answering questions I hear most often.

What should I expect from a treatment for SMA?

Everyone’s situation is different, so I like to ask my patients about what their treatment goals are. I’ll ask if there’s a specific function that they have today that’s most important, like using their wheelchair or being able to eat on their own, and whether they’re hoping to try to improve their skills or maintain what they have at the moment. Then I usually talk through what’s realistic based on their age and current level of function, what we know about the potential benefits and risks from the clinical trials, and how we’ll track their specific results over time. So much of what to expect is based on an individual’s goals. It’s important for me to find out what matters most of the specific patient or family and then map out a plan from there. I also like to let my patients know about any support programs that may be available for them. We also discuss the potential risk of not treating, and this is a very valuable conversation. It’s important to talk to your doctor about what’s realistic for you, as well as any side effects you could experience in any existing medications that may need to be adjusted, as all of these elements can impact a person’s experience when starting treatment.

Will Evrysdi work for me?

That’s a great question that I get asked a lot, but no doctor can predict whether a medication will work for everyone or what that may look like in each person. Clinical trials provide important information about how the medicine impacted other people to help us to decide if it could be a good fit for you. Evrysdi was studied in more than 490 people with SMA. The results from these clinical trials show that Evrysdi had meaningful benefit for adults, children, and infants with SMA. In adults and children with Type 2 or 3 SMA, Evrysdi significantly improved or maintained motor skills compared with placebo. This was measured by the Motor Function Measure-32 item scale, or the MFM-32 for short. In infants with SMA Type 1, Evrysdi help them to achieve a key motor milestone and delayed disease progression. Evrysdi was also studied in infants with presymptomatic SMA. The decision to go on treatment is a personal one that should be made with you and your doctor.

Are there any side effects?

This is such an important question. When considering a new treatment, it’s important to weigh any possible side effects against the potential benefits of treatment. It’s also important to speak to your doctor about any risks, so it’s a good discussion to have with your doctor in advance, not only to weigh the potential risks and benefits but also to prepare yourself and understand what to expect. The safety of Evrysdi was established over multiple trials that included more than 490 people with SMA Types 1, 2, and 3, as well as infants with presymptomatic SMA. These included people with a broad range of physical ability, including those who could walk in and those who could not, people with and without severe scoliosis or joint contractures, and those who have previous treatments with SMA medications which included approved or investigational SMA therapies. It’s important to talk to your doctor about any other conditions or medications that you may be taking as they can impact effects and side effects, as well as to create a plan for how you might manage any that you may experience. Here, we can see the most common side effects seen in the Evrysdi clinical studies.

I’ve seen on social media that some people are getting diarrhea. Will that happen to me? If so, how long will that last?

This is one of the questions I get asked the most. Social media is a great place to connect with the community members, but it isn’t an appropriate place to get medical advice, especially since everyone’s personal experience can be different. It’s important to ask your doctor these kinds of questions and to discuss the clinical trial results with him or her to get a sense for what you might expect based on that data. In the Evrysdi clinical trial of adults and children with SMA Types 2 and 3, 17% of people taking Evrysdi experienced diarrhea compared to 8% of the people who were taking placebo in the first year of treatment. Of those who did experience diarrhea in the first year of treatment in the study, 80% resolved within the week, with 12% of cases lasting longer than 2 weeks. With all of my patients, I review all medications they’re taking before starting Evrysdi, including vitamins and over-the-counter supplements. For instance, if I have a patient who typically takes a laxative, we talk about whether we should adjust that. We review how and when to take Evrysdi, for example, after a meal, then followed by water. We make a plan to connect immediately if they do experience any side effects so we can determine the best way to address them together.

I know Evrysdi increases protein levels, but will those levels go up and down?

Ah, that’s a complicated one. Okay, to answer this question, let’s first talk about what happens in the body of someone with SMA. The human body needs a key protein called the survival motor neuron protein, or the SMN protein, for muscles to function properly. The SMN protein has several important jobs within the cells that ultimately enable our bodies to move. It is found at all cells and all tissues in the body. People with SMA have low levels of this protein, causing their muscles to break down, and this has a widespread impact from head to toe. Evrysdi is designed to help the body make more of the SMN protein and keep the levels up. In clinical trials, within four weeks of treatment with Evrysdi, the SMN protein levels in the blood more than doubled. And these levels were maintained for all of the SMA subtypes throughout the month of the studies. Based on these trial results, we expect Evrysdi to have a sustained impact on the SMM protein levels when taken correctly.