<div>Hello,
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<div>My name is Liam.
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<div>Before I was 3 months old, I found out I had Niemann Pick Type-C (NPC), a rare fatal neurodegenerative disease. This disease progresses very rapidly; most kids with NPC pass away before they are 10. My mom had a cousin who passed away when she was 4 because she couldn't get any treatment. NPC has no cure right now, and FDA has not approved any treatments that could help me live longer. I will be lucky if I can live to be 5 years old...
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<div>It's been over a year since my doctors confirmed I have this terrible disease, and I have not been able to access any experimental treatments that could help me have a chance at life. I have been told that I am not sick enough yet, according to the FDA. I have to wait and let the disease build up first. I feel like hope is just out of reach. Many drugs currently in clinical trials have proven to prolong the life of children who receive them early enough. My disease is very complicated, and there aren't many in the world like me, so scientists don't have a lot of data for NPC to show the FDA. You'd think they'd want us Rare Kiddos to stick around longer to understand NPC better and make more data.
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<div>I need help to get treatment early and make treatment options available for other Rare Kiddos with NPC like me. Anything you donate will help take care of me, help pay for research to cure NPC, and help advocate for better federal regulations that allow children born with rare fatal diseases to get treatment sooner.
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<div>Thank you,
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<div>Liam
</div><img alt="" media_type="3" src="https://d2g8igdw686xgo.cloudfront.net/69009423_1673360611590077_r." width="100%" />
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<div>**Update Dec 2022**
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<div>I have finally been able to start an experimental treatment under a sub-study of Arimoclomol, see below for more.
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<div>~~~
</div><img alt="" media_type="3" src="https://d2g8igdw686xgo.cloudfront.net/69009423_1673360232672051_r." width="100%" />
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<div>When Liam was born in the Summer of 2021, he showed signs of liver complications that caused him to spend his first week in the NICU. After that week, doctors were unable to find anything that explained why Liam’s liver levels were getting increasingly worse day after day.
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</div><img alt="" media_type="3" src="https://d2g8igdw686xgo.cloudfront.net/69009423_1673360275770098_r." width="100%" />
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<div>Months of weekly blood draws and regular follow-up visits to specialists and pediatricians still did not reveal the cause of Liam’s poor health condition. In August, Liam’s health began to rapidly decline, forcing us to take Liam to the ER at Primary Children’s Hospital. The medical teams there were able to stabilize Liam’s health, treat a viral infection he had caught and perform additional testing. After another week in the hospital, we made our way home again. A few weeks later the doctors from PCH called with the results from genetics testing and gave us the terrible news of our son’s diagnosis of Niemann Pick Type-C.
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<div>Since the day we learned Liam had NPC, his doctors started searching for treatment options. There are no known cures for NPC and only a few experimental treatments that slow the progression of the disease with varying levels of success.
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<div>The current “Standard of Care” for a person with NPC is a drug not approved by the FDA to treat NPC called Miglustat. This drug is FDA approved to treat Gaucher's Disease, another lysosomal storage disorder. In 2018, Miglustat ranked in the top 20 most expensive drugs in the world. Today, it can still cost over $300 per 100mg pill. Miglustat has been shown to help slow the progression of NPC disease, especially when used in combination with other experimental drugs. We are grateful to our doctors who helped push for Liam to receive Miglustat at 6 months of age.
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<div>There are some experimental drugs undergoing clinical trials for NPC. The drugs range from oral medications, like Arimoclomol, to direct injections into the spinal fluid, like Cyclodextrin. While NPC is a complicated disease that impacts every cell in the body, doctors and scientists focus on treating the brain and the nervous system since they are the most sensitive and crucial to preserving life.
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<div>Unfortunately, just being diagnosed with a fatal genetic condition is not enough to qualify for a clinical trial of medicines that have been shown to prolong life with the disease. From the day these children are born this disease is working against them, it is not a matter of if the disease with take their lives, it’s only a matter of when. Generally, a family will struggle for years trying to figure out what is wrong since NPC is often misdiagnosed until it has become severe and it’s too late to treat. Liam was diagnosed with NPC uncommonly early. A huge blessing since the earlier you can start treating NPC the longer you can delay the neurological onset of the disease.
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<div>We have been working with doctors to get Liam on any experimental treatment that we can as soon as possible. Liam has been too young and not severely sick enough to meet the required criteria to be granted compassionate use or early access to any of the ongoing trials. Time has been painfully ticking away as each avenue we pursued led to a dead end.  A ray of hope came in April of 2022 when we learned one of the clinical trials for Arimoclomol was planning to open a sub-study for children 6 months to 2 years of its oral medication. This drug, Arimoclomol, has been shown to slow the progression of NPC when combined with Miglustat if the patient has gene mutations that respond well to the medication. Some NPC gene mutations do not seem to respond at all to Arimoclomol. Liam’s gene mutations have the potential to respond to Arimoclomol so we are hopeful it can help buy precious time. For 8 months we anxiously waited for the trial to get FDA &amp; IRB approval. In early December of 2022, it finally came through and Liam was allowed to start taking Arimoclomol.
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<div>Even with Liam taking both Arimoclomol and Miglustat, we know that the clock is still ticking for him. We pray that the combined effect of these two drugs will slow the disease enough for better medications to be developed that can cure or make the disease chronic.
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<div>Along with helping pay for Liam’s medical expenses, we have set a goal with this GoFundMe to raise $100,000 to pay for initial medical research of using mRNA to treat NPC. If the testing and research go well we will continue to raise funds to push the treatment to clinical trials.
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<div>We are extremely grateful for all the kindness &amp; love that we have received and continued to receive.
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<div>Thank you,
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<div>Liam, Jennifer &amp; Jordan Mitchell
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<div>Venmo: @JordanAMitchell
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<div>Learn More about Liam &amp; his Fight against NPC at <a href="https://www.life4liam.com" target="_blank" rel="noreferrer noopener">Life4Liam</a>.com and <a href="https://www.rarekiddos.com" target="_blank" rel="noreferrer noopener">RareKiddos</a>.com
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Life 4 Liam: Fight Against NPC

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Life 4 Liam: Fight Against NPC

Still $155,581 to go. Help us build momentum.