“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 3)

In the first part of the “Save Our Medicine” series, you met moms Tiffany, DeAnna, and Sara, who are working to make a change and save experimental medication access for families with NPC. Next, in part 2, we discussed adrabetadex, genetic testing, and the dangers of waiting for treatment. Join us now, in the final part of the “Save Our Medicine” series, to discuss the movement as a whole, the recent “You Are My Sunshine” video, and how you can help save experimental medication access.

To read part one of the series, head here. For part two, head here.

Save Our Medicine

So how can we help to amplify these stories and work to save medicine for families with NPC? Tiffany, DeAnna, and Sara began speaking to their friends in similar situations whose children were on expanded access and responded well to the drug. They thought about making a podcast, speaking to doctors, or using online platforms to raise awareness.

They decided to streamline some of their social media to create the Save Our Medicine website. You may use the website to donate to the campaign, sign a petition advocating for increased access, or learn more about the families affected.

The mothers stressed that Save Our Medicine is not just about adrabetadex. Sara says:

This is the medicine that our kids take and that they need, but we want all of them. There are over 300 known NPC mutations, and the medicines are not interchangeable. Some work for some kids and some work for others. We want medicine for the whole spectrum, not a one-size-fits-all. As a parent, we don’t have a voice. We want to get different therapies that can help your child because you know your child.

That’s one of the core focuses of the movement: giving parents a voice. Doctors are medical experts, and parents are the experts in their children.

“You Are My Sunshine”

If you teared up while watching Save Our Medicine’s “You Are My Sunshine” video, you’re not alone (believe me, I did too). This heartfelt and powerful video features nine mothers singing the song to 13 of their children. DeAnna and Osama, Sara and Marian, and Tiffany, Mason, Logan, Ethan, and Jacob (in photo) are all featured. The video, developed for NPC Awareness Month and to work to save life-sustaining medications, was also shared online by Jana Kramer, a country music artist and activist teaming up with the Hope for Marian Foundation.

How You Can Help

These mothers are not alone in their fight. It’s time for us, and the medical community, to stand behind NPC families and get them the help and medication access that they need. During the global pandemic, the medical community quickly mobilized to get vaccines rolling out. In the same way, the medical community can exercise regulatory power to help the NPC community.

This isn’t a matter of making a choice between medication access and research towards a cure. Both of these things can feasibly happen at the same time. That being said, it is incredibly important to listen to the voices of those affected, those who are living this day in and day out. To hear Sara, DeAnna, and Tiffany explain how their children have been positively affected by treatment. To learn how Emma Peterka can now sit up on her own thanks to her treatment. To understand that these are children, not statistics. Every adult or child with NPC has family, friends, teachers, and a community who loves them. We need to be heroes for them. We should not be fighting for common sense. As Sara says:

We need help now, not in 6 months. Please don’t make us jump through hoops.

If you’d like to use your voice to Save Our Medicine, you can do the following:

  • Donate to the campaign at Save Our Medicine. Every dollar counts.
  • Sign and share the org petition, which is now over 46,000 signatures!
  • Share this story, or other NPC stories, on social media. Make sure to use the hashtags #NiemannPick, #NPC, and #SaveOurMedicine.
Save Our Medicine is fighting to save medication access for families with NPC
Courtesy of: Save Our Medicine (FB Page)
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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