Lessons Learned From Unanticipated Positive Outcomes

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by Kelly Wallis |

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This is a follow-up to my previous column about my 26-year-old daughter, Abby, potentially being accepted into a clinical trial.

To recap, my husband and I were recently notified that she might be accepted into a clinical trial for a drug called Kineret (anakinra). Researchers will study the drug’s effectiveness in mitigating certain symptoms of Sanfilippo syndrome, a terminal, degenerative disorder that’s within the family of lysosomal storage diseases.

We were initially very excited about the possibility of getting into the trial and maybe finding some relief for Abby (and us as caregivers). But traveling with a disabled adult comes with challenges.

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When it came time to plan the trip to California from Texas, we were filled with anxiety. At first, we thought we would fly, but considering the ongoing pandemic and all the necessary safety protocols in place, we weren’t sure Abby could handle it. We also didn’t want to be at the mercy of the airline if Abby did have trouble during the trip, such as not wearing her mask or having an accident.

So, we drove — for two and a half days. It was me, my husband, Abby, and her caregiver, Aly, loaded up in the van. I am happy to report that Abby was an incredibly good passenger for almost the whole trip!

One thing to know is that Abby never naps. She stopped napping when she was 2 years old. This was a disadvantage in that we felt the need to keep her happy or occupied almost all the time. However, thanks to iPads with downloaded movies, and phones with Spotify playlists of songs she loves, we were able to keep her content for most of the time.

She was a trooper through all the restroom stops and five nights of sleeping in different hotels. (Some scenes from the movie “Rain Man” would come to mind when we arrived at a new hotel.) Most nights, she slept OK. At the actual clinic appointment in California where researchers got all the assessments and measurements they needed from Abby, she did a great job and tolerated everything amazingly well.

The trip taught us several things, but the most important outcome is that we are grateful. We are grateful to the Cure Sanfilippo Foundation for funding the trial. We are grateful that Aly agreed to go with us, as she was a huge help. And we are grateful for the opportunity to be included in the trial, especially at Abby’s age. Most clinical trials for Sanfilippo syndrome don’t accept participants over a certain age, which we understand. Once diagnosed with Sanfilippo, kids rarely live into their teens, so the research must focus on the younger ones.

What started out as anxiety about potential pitfalls and unknowns turned into unexpected relief and the realization that we are able to travel with Abby. She can handle it.

Don’t misunderstand, it took lots of patience to physically manipulate a grown adult to go where we wanted her to go and help her get in and out of the van countless times. It was hard, but we did it, and we can do it again when the time comes. So, it was a win for us.

I will continue updating once we are, hopefully, officially accepted.


Note: Sanfilippo News is strictly a news and information website about the syndrome. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Sanfilippo News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Sanfilippo syndrome.


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