Jordan

Father of @Life4Liam diagnosed with Niemann Pick Type-C. Fighting for a Cure. Donate at

[Life 4 Liam & Friends @Life4Liam]

Individually rare, collectively as common as Childhood Cancer - but no one knows it exists.
On Childhood Dementia Day we wanted to shine a light on some of the brave, beautiful children with dementia, and the heartbreaking conditions they are battling.
Over 50,000 babies are born with one of the 145 different genetic conditions that cause Childhood Dementia every year.
All of these conditions are terminal, taking a life every 11 minutes, with an average life expectancy of just 10 years old.
Childhood Dementia is progressive. Children are often born apparently healthy then over time lose the ability to walk, talk, run, play, eat, drink & eventually their lives.
It's cruel, relentless & devastating.
There are no approved treatments for many Childhood Dementia-causing conditions anywhere in the world & over half have no active clinical trials.
We are fighting to change that, and together we can save these brave, beautiful children. Please share!

[Fei-Fei Li @drfeifei]

Agents can be AI, but agency is human. It is every individual’s responsibility to empower our own agency, with the help of tools. But the North Star should always remain human centered.

[FDA Drugs and Biologics @FDADrugs]

Today, the @US_FDA approved the first treatment for ataxia-telangiectasia (A-T) in adults and pediatric patients weighing at least 15 kg, reflecting an important milestone for families facing this progressive condition and our continued commitment to patients with rare diseases. https://t.co/kXVZwuP7zK

[Life 4 Liam & Friends @Life4Liam]

Beren Therapeutics launched Test for NPC: disease education plus NPC GenomeComplete — no-charge whole-genome sequencing (GeneDx), family testing, and genetic counseling for eligible patients. Preliminary results in about 48 hours for children in rapid neurological decline.

They say roughly two-thirds of people with NPC in the U.S. are still undiagnosed. If a clinician is thinking NPC, this is one path to answers.

https://t.co/poOsFkzGAO

[Schultz_PhD @M_Schultz_Lab]

Check out our recent publication, also Ellen made the cover image!: Codon-optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo: Molecular Therapy Nucleic Acids https://t.co/roScSvPovB

[Jordan @JordanAMitchell]

Find the paper from Schultz Lab here:

https://t.co/HmHstNc07J

[Jordan @JordanAMitchell]

The September issue of Molecular Therapy Nucleic Acids is out, and the cover is ours to celebrate: Ellen Koufer’s art, Schultz lab science, work @Life4Liam helped fund.

[Life 4 Liam & Friends @Life4Liam]

The September issue is here.

Our Iowa gene-therapy paper is in Molecular Therapy Nucleic Acids, Vol 37 Issue 3. Codon-optimized Npc1 mRNA in lipid nanoparticles. First in-vivo validated mRNA/LNP path for NPC1.

Open access: https://t.co/wY8Axtt8Am

#NPC #NiemannPickDisease

[Life 4 Liam & Friends @Life4Liam]

In mice missing NPC1, one IV dose got the liver making the protein again. Autophagy came back toward normal. Lipid and liver-injury markers improved.

Keep dosing, keep the protein. A functional-cure path. Not the finish line.

https://t.co/8ch9CoNVTH

#CureNPC #NiemannPickDisease

[Life 4 Liam & Friends @Life4Liam]

The September issue is here.

Our Iowa gene-therapy paper is in Molecular Therapy Nucleic Acids, Vol 37 Issue 3. Codon-optimized Npc1 mRNA in lipid nanoparticles. First in-vivo validated mRNA/LNP path for NPC1.

Open access: https://t.co/wY8Axtt8Am

#NPC #NiemannPickDisease

[Jordan @JordanAMitchell]

Excited this work is finally published. The effectiveness of this gives us so much hope that we will save our son and children with NPC.

Very grateful and proud of everyone who played a part and helped prove this is a path to a cure.

There has been a lot of great developments since this initial phase completed as we prepare for the next phase of testing it in the brain.

[Life 4 Liam & Friends @Life4Liam]

We have been waiting to share to this.

The first phase of gene-therapy work our families helped fund at Schultz Lab and the University of Iowa Stead Family Children’s Hospital is published. In mice with NPC, they delivered the instructions for the missing protein. The liver started making NPC1 again.

Keep giving the doses, keep the protein. A functional-cure path. The start of the right road to a true cure.

Read it here: https://t.co/LBZ29F3nai

[Life 4 Liam & Friends @Life4Liam]

We have been waiting to share to this.

The first phase of gene-therapy work our families helped fund at Schultz Lab and the University of Iowa Stead Family Children’s Hospital is published. In mice with NPC, they delivered the instructions for the missing protein. The liver started making NPC1 again.

Keep giving the doses, keep the protein. A functional-cure path. The start of the right road to a true cure.

Read it here: https://t.co/LBZ29F3nai

[LVMPD @LVMPD]

BREAKING

An officer-involved shooting occurred on August 4th, in the 4500 block of East Tropicana Avenue. This shooting left a suspect deceased at the scene. An LVMPD officer was transported to a local hospital, where, despite all life-saving efforts, he passed away.

The officer had been with LVMPD since November 2023. Additional details will be released as they become available.

Our condolences go out to the officer's family. We will support them in every way we can in the days ahead.

This incident highlights the dangers law enforcement officers face in the line of duty.

[Zevra Therapeutics @zevratx]

Now published in Molecular Genetics and Metabolism Reports: “Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy.”
Read our press release to learn more https://t.co/O8nzz41dX6

[All day Astronomy @forallcurious]

🚨: The most important sky events of this decade is occurring on February 28th. 🌌 ✨

Six planets will align and put on a show of our lifetime. Mercury, Venus, Jupiter and Saturn will be visible to the naked eye from almost anywhere.

Uranus and Neptune will be visible to naked eye if seen from dark places.

MARK YOUR CALENDERS; February 28th. 📅✨

[A Man Of Memes @RickyDoggin]

Mark Your Calendars !

FEBRUARY 28, 2026
Don't forget to look up the planets will drift quietly across the sky, reminding us how beautifully the universe can align.

[Elon Musk @elonmusk]

!!

[Cyclo Therapeutics, Inc. @Cyclodexpert]

Niemann-Pick is a rare, inherited lysosomal storage disorder impacting both children and adults. Discover how we’re striving to develop potential treatments for this critical unmet need.

https://t.co/osRXB2QmH7
$RFL #NiemannPick #NPC

[Life 4 Liam & Friends @Life4Liam]

Our fight for Liam and others with Niemann-Pick Type C is at risk due to proposed $18B NIH cuts. Research is our hope for a cure. Read our story and join us to advocate for funding! #NPC #RareDisease #SaveResearch

https://t.co/Nq1A8N53O0

[Life 4 Liam & Friends @Life4Liam]

Prime Editing for Chronic Granulomatous Disease (CGD)

Prime Editing restored 66% immune function in a CGD patient—3x above curative threshold. Insights like this could inspire treatments for lysosomal disorders like NPC. Hope for rare diseases! #RareDisease #GeneTherapy #NPC

[Life 4 Liam & Friends @Life4Liam]

Exciting new review on advancing mass spectrometry in lipidomics for Niemann-Pick type C (NPC) disease! Explores MS tools, biomarkers like oxysterols & sphingolipids, and future integration with AI & omics for better diagnosis & therapies. A must-read for rare disease research!