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Nine patients with an ultra-rare type of ALS have been treated during the first year of a multi-year NIH grant. Silence ALS aligns Columbia Medicine and n-Lorem on the mission to develop personalized experimental ASO medicines to treat ultra-rare ALS patients. bit.ly/4icjqOf

Our bodies are like bustling cities of cells, always chatting and working together. They don’t just send long-distance messages to organs—they also gossip locally, getting nearby cells to spring into action. Stream Part 2 of Cell Communication: nlorem.org/how-cells-phon…

We're proud to recognize all individuals living with CACNA1A! Caused by a mutation of the CACNA1A gene, it often leads to epilepsy, episodic ataxia, hemiplegic migraines, atypical eye movements, developmental delays, and autism spectrum disorder.

For n-Lorem’s Chief Operating Officer, Sarah Glass, PhD, the mission of n-Lorem is deeply personal. Sarah's son, Ethan, was diagnosed with a nano-rare mutation, leading her family on a daunting journey and ultimately shaping her commitment to the cause. youtu.be/xXNx9traZE8

𝓜𝓸𝓶 𝓷𝓮𝓮𝓭𝓼 𝓪 𝓫𝓻𝓮𝓪𝓴 The n-Lorem Mother’s Day Candle & Card Fundraiser is here! Pre-order a thoughtful gift that says "I love you" and "take five, Mom" all in one. All proceeds support n-Lorem's mission. nlorem.org/mothers-day-ca…

n-Lorem Foundation Celebrates Five Years of Bringing Hope and Potential Treatments to Nano-rare Patients Press release: nlorem.org/n-lorem-founda…

In 2024, Connor received his personalized ASO medicine, and while Dr. Kim-McManus of Rady Children's continues to fine-tune the optimal dose and schedule, Connor has, for the first time, taken steps on his own and experienced periods without seizures. youtu.be/egN2j2vrMEo

Have you thought about Mom? 💛 Give a gift that does more than smell amazing (hello, lemongrass!). The n-Lorem Candle & Card Fundraiser offers a meaningful way to celebrate the moms who light up our lives—while supporting free, lifetime treatments nlorem.org/mothers-day-ca…

We're proud to recognize all individuals living with KIF1A-Associated Neurological Disorder (KAND)! To learn about KAND and their superheroes, visit kif1a.org KIF1A.ORG

The journey to a rare disease diagnosis can span weeks, years, or even decades—and for some, a diagnosis may never come. Today, we honor all individuals and families still searching for answers, as a diagnosis is the crucial first step toward finding a potential treatment.

Freshen up on ASO Technology 〰️🧠 Did you know? n-Lorem has released over 30 Intro to Medical Science episodes breaking down complex topics—like ASOs, risk assessment, and advanced genetics—to help patients and families make informed decisions. youtube.com/playlist?list=…

Today marks the first-ever TUBB4A Awareness Day! TUBB4A-related leukodystrophy affects the brain’s white matter. Caused by a mutation in the TUBB4A gene, symptoms include spasticity, seizures, and swallowing & speech issues. Here are a few faces of those fighting TUBB4A!

May is ALS month, and we’re honored to co-found the Silence ALS initiative, which aligns Columbia University, Dr. Neil Shneider, and n-Lorem on the joint mission to develop personalized experimental ASO medicines to treat ALS patients with “nano-rare”, pathogenic gene mutations.

Research, published in JCI Insight and supported by the Wolverine Foundation, identifies new and important roles of JIP3 — offering critical insights into therapeutic approaches for nano-rare patients. shorturl.at/pKBSa

⚗️ Understanding the basics helps patients make more informed decisions about their care—and helps scientists design better treatments. Biological Systems & Chemical Networks: youtu.be/rg1_sHPlswg Intro to Medical Science Playlist: youtube.com/playlist?list=…

Last week, members of our team, including pre-clinical development senior scientist Catherine Parisien, presented a poster on the discovery and development of individualized ASOs for those with ultra-rare forms of ALS at the Target ALS Annual Meeting in Boston.

The NFLP assay, launched by Mayo Clinic, has helped assess the impact of an n-Lorem-discovered and developed treatment for a patient with CHCHD10-related ALS. news.mayocliniclabs.com/2025/04/14/neu…

Induced pluripotent stem cells (iPSCs) are a groundbreaking, and mind-blowing, scientific advancement—one of many that, without them, n-Lorem would not be able to do what we do. nlorem.org/what-are-ipscs…