UNC Neurology is proud to be a clinical site within the national n‑Lorem Foundation nano‑rare patient research and treatment network, an innovative initiative advancing personalized therapies for patients with ultra‑rare genetic neurological disorders.
The n‑Lorem Foundation, a nonprofit established in 2020, is dedicated to discovering, developing, and providing individualized antisense oligonucleotide (ASO) therapies—at no cost and for life—to patients with “nano‑rare” mutations, defined as those affecting fewer than 30 individuals worldwide. These conditions often lack approved treatments, leaving patients and families with limited options.
As a participating clinical site, UNC plays a critical role in the evaluation, treatment, and long‑term monitoring of patients receiving these bespoke investigational therapies. The program brings together experienced clinician‑scientists, advanced genomic analysis, and rigorous safety oversight to ensure that each patient’s treatment plan is both scientifically sound and patient‑centered.
In a recently published perspective in Nucleic Acids Research, investigators detailed the n‑Lorem model and early clinical experience, reporting encouraging signs of clinical benefit across multiple neurological conditions, alongside a favorable safety and tolerability profile. Neurological disorders make up the majority of conditions treated through the program, underscoring the importance of specialized neurology centers like UNC in delivering this highly individualized care.
Addressing the needs of nano-rare patients: the n-Lorem experienceUNC’s participation in the n‑Lorem network reflects the department’s ongoing commitment to innovation, collaboration, and expanding access to cutting‑edge therapies for patients with the greatest unmet needs. Through partnerships like this, UNC Neurology continues to advance precision medicine while offering hope to families affected by the rarest of diseases.