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My 4-year-old daughter, Elle, is in the fight of her life. Her older sister, Milla, lost her fight last November. Their opponent? Batten disease, a rare, fast-moving, and fatal condition that destroys the central nervous system’s ability to function.

Elle has a chance to help manage, or maybe even beat, her disease that Milla didn’t have: a clinical trial in which an investigational protein is infused directly into Elle’s brain every 14 days.


As I have watched Elle bravely confront Batten disease, I have also been following discussions within President Trump’s administration about potential changes to the Food and Drug Administration. Some of these are highlighted in a STAT article, others in the president’s first speech to Congress, which just happened to be delivered on Rare Disease Day. I can’t help but wonder how the administration’s desire to streamline the FDA might affect my family, which has already been irrevocably changed by a rare and devastating disease.

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  • I agree with Frazer Giesselmann. It’s crucial not to bog down the research in rare diseases especially if the parents are desperate because there are no other options for their children. What’s to lose?

  • Way to not mention the changes to the FDA in the past decade; orphan drug status, accelerated drug approvals, and concurrent stage studies with small cohorts to push efficacious treatments through faster. I feel for the family, but safety and efficacy has to be proven at some level.

  • Dear Frazer and Dana, That was very well written and I wish that President Trump, etc al could read this. I am so sorry about all of what you have gone through with your daughters. Love from your cousin, Nancy

  • Thank you for writing this. Our sweet six-year-old Mila was just diagnosed with Batten Disease. The connection between our two Mila/Milla is so powerful. We are devastated, but are fighting hard to start our own clinical trial as quickly as possible as none exists for her type. We are hoping that the FDA understands just what you wrote – that the process needs to be quick in order to give children with rare diseases a chance at life. So thank you for telling your story and opening up about your pain in order to help other families like ours.

  • Very well written Frazier. ECS English teachers should be proud. Very moving article that brought tears to my eyes your girls are heroes and so is your entire family. We love you and pray for you all continuously. Hope someone knows how to send this to Trump. !!!!!!

  • What a heartbreaking and at the same time full of hope story. I teach genetics and one of topics I cover is rare genetic mutations and what are some of the issues with FDA approvals and the horrific cost both emotional and financial for the families. Awareness and changes in the way we fund discoveries are very much necessary. In today’s political climate I find that we are looking at a long arctic winter.

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