n-Lorem Foundation

Now streaming on CNBC Cures: Defying Rare Disease—see the stories and breakthroughs bringing hope to millions. cnbc.com/video/2026/03/…

Defying Rare Disease premieres tonight on CNBC at 7 PM ET which follows families navigating rare diseases alongside the scientists, advocates, and innovators working that features familiar faces, including KIF1A patient Susannah and n-Lorem founder Dr. Stan Crooke!

Today we stand with the CACNA1A community and the scientists working toward answers. CACNA1A -related disorders are rare genetic conditions that can affect the brain and nervous system. #CACNA1A #RareDiseaseAwareness #RareDisease #ScienceForPatients

Hope for More and Dream Bigger! Check out our new nlorem.org homepage(s). Refresh the page a few times to see different faces 🧡

Someone out there needs a lucky charm. 🍀Donate today and help fill the Pot O' Gold to bring personalized treatments to patients with nano-rare genetic diseases. nlorem.org/donate/

Watch: Full CNBC Cures Summit panel discussion featuring n-Lorem founder, Dr. Stan Crooke - cnbc.com/video/2026/03/…

New Podcast Episode 🎙️ Andrew Lo, Ph.D. joins the show to explore where the emerging FDA draft guidance may lead for nano-rare, as well as discuss the economics of rare disease drugs, including the estimated cost of care for a rare patient. youtu.be/DCue7Dy9HoE?si…

It’s KCNQ2 Awareness Week, and we are proud to recognize, celebrate, and channel hope for individuals living with KCNQ2. This rare genetic condition, caused by a mutation in the KCNQ2 gene, can lead to severe epilepsy, hypotonia, spasticity, and developmental delays.

Establishing a commercial solution for extremely rare genetic diseases - @DrStanleyCrooke @AndrewWLo go.nature.com/4aHSIvu

It’s #RareDiseaseDay and the path forward is brighter when we walk it together. Patient families, physicians, our team, friends, and loved ones came together to Chalk the Path Forward, filling our office sidewalk with messages of hope and awareness. nlorem.org/donate

Rare is more common than you think. Ahead of #RareDiseaseDay, we stand alongside the tens of millions of patients and families worldwide living with rare diseases. n-Lorem is proud to push science forward for this community so that we can hope for more and dream bigger together.

The first-ever CNBC Cures Summit on March 3rd will spotlight conversations focused on accelerating the future of medicine through breakthroughs in rare disease research. Learn more and register for free here: bit.ly/4cinix3 #CNBCCures

International SCN2A Day is observed on February 24th because the SCN2A gene is located on the long (q) arm of chromosome 2 at position 24.3. The SCN2A gene provides instructions for creating a sodium channel protein vital for brain cell communication. #SCN2Aawareness

Reflections from the n-Lorem Chairman | 2026 Dear Patients, Families, Supporters, and Colleagues, In this video message, Chairman Stan Crooke reflects on n-Lorem, six years after founding. We invite you to watch: youtu.be/AvMW363ES4E

PACS1 Awareness Day is observed on February 7, honoring the birth date of the first individual ever diagnosed with the condition. To learn more about PACS1, visit pacs1foundation.org and pacs1smiles.com #PACS1Awareness

No matter who you root for on Sunday, you can score a win for patients today with a donation 🏈 nlorem.org/donate/

This rare genetic condition, caused by a mutation in the #ASXL3 gene, can involve developmental delays, low muscle tone, intellectual disability, limited or absent speech, feeding challenges, constipation, behavioral and sensory differences, dental abnormalities, and seizures.

Today, we're launching a NEW SERIES that focuses on the scientific and medical breakthroughs that make n-Lorem possible. We call them miracles and without them, n-Lorem would not exist today. Episode 1 🔗: youtu.be/nSAHD8SkZcA

Rare Disease Month reminds us families are searching for answers every day. #PacBio is collaborating with @n_lorem and Esperare to advance precision therapies for people with ultra-rare genetic diseases. Families deserve more than hope. 🔗bit.ly/3NsBCbH #WeCareForRare

Article: cnbc.com/2026/01/30/rar… Podcast: squawk-pod.simplecast.com/episodes/the-p…

A new CNBC Cures article and The Path with Becky Quick podcast episode are out now, discussing the realities of Susannah’s disease and highlighting our work to deliver personalized ASO medicines to patients who previously had no options.