{"id":14907,"date":"2026-06-02T22:34:00","date_gmt":"2026-06-02T20:34:00","guid":{"rendered":"http:\/\/stocks-future.com\/?guid=193986d7fdb9ac1ac7093a8b0ed9d05e"},"modified":"2026-06-02T22:34:00","modified_gmt":"2026-06-02T20:34:00","slug":"new-publication-provides-an-unprecedented-look-at-the-largest-reported-cohort-of-nano-rare-patients-evaluated-for-individualized-aso-therapies","status":"publish","type":"post","link":"https:\/\/stocks-future.com\/?p=14907","title":{"rendered":"New Publication Provides an Unprecedented Look at the Largest Reported Cohort of Nano-Rare Patients Evaluated for Individualized ASO Therapies"},"content":{"rendered":"

\nFirst-of-its-kind perspective from the n-Lorem Foundation provides a very broad review of n-Lorem\u2019s experience in more than 300 patient applications and reports excellent safety profile and meaningful clinical benefit in treated patients receiving individualized ASO therapies<\/i><\/p><\/a><\/a>

SAN DIEGO--(BUSINESS WIRE)--#ASO<\/a>--n-Lorem, a nonprofit foundation that discovers, develops and provides personalized experimental antisense oligonucleotide (ASO) medicines to patients with nano-rare mutations (1-30 known worldwide) for free, for life, announced today the publication in Nucleic Acids Research [https:\/\/doi.org\/10.1093\/nar\/gkag504<\/a>] that details its experience with the first 329 nano-rare patient applications for treatment and a summary of the results to date in nano-rare patients treated with experimental ASOs medicines provided by n-Lorem.<\/p>

\nn-Lorem has created the largest safety database of nano-rare patients treated that includes more than 50 nano-rare patients treated with bespoke ASO medicines discovered and developed at n-Lorem, more than 300 doses administered across all routes of administration (subcutaneous, intravitreal, intrathecal), and more than 55 patient years of safety data. Amongst treated patients, one patient has been treated for longer than three years, six patients for longer than two years and 26 patients for more than one year. In all treated patients, n-Lorem has observed an excellent safety and tolerability profile with no ASO-related serious adverse events.<\/p>

\nn-Lorem also highlighted the meaningful impact of its ASO medicines on individual patients. n-Lorem is observing improvements observed in treated nano-rare patients that include, significant reductions in seizures, substantial improvements in control of movements, reduction of neuropathic pain, improved autistic symptoms, recovery of development delays that were lost and acquisition of new skills such as walking independently, improved quality of life based on pre-defined endpoints and standard measures, stabilization of disease in ALS patients, halting of vision loss, and halting loss of kidney function.<\/p>

\n\u201cAt n-lorem we are treating more than 50 nano-rare patients for free, for life and adding more patients every month. The systems and processes we have developed and the broad expertise in ASO technology has meant that we can meet our commitment to treat our patients with safe and effective ASOs,\u201d said Stanley T. Crooke, Founder, Chairman and CEO of n-Lorem. \u201cWe are also thrilled with the benefits we are observing in nano-rare patients with disorders that manifest in the CNS, retina, and the kidney. Many of our patients begin treatment with very advanced diseases, many that have missed key developmental milestones, yet we continue to observe significant improvements in these patients, some responding even at very low doses. These observations demonstrate that many developmental delays can be recovered even a decade or more after the normal age of achievement of the milestone. Moreover, we show that patients can achieve milestones never achieved.\u201d<\/p>

\nThe Nucleic Acids Research publication [https:\/\/doi.org\/10.1093\/nar\/gkag504<\/a>] titled \u201cAddressing the Needs of Nano-rare Patients: the n-Lorem Experience\u201d provides a comprehensive overview of the processes and systems that n-Lorem has industrialized that introduce efficiencies across the entire organization, highly sophisticated design algorithms that are a product of nearly 40 years of experience in creating, validating and advancing the technology, and scaling to meet a larger demand while maintaining high-quality standards. These processes include:<\/p>