N-lorem Foundation
Clinical trials sponsored by N-lorem Foundation, explained in plain language.
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Custom drug targets rare ALS in first-of-its-kind single-patient trial
Disease control Not yet recruitingThis study tests a custom-made antisense oligonucleotide (ASO) designed for one person with ALS caused by a specific CHCHD10 gene mutation. The goal is to slow disease progression by targeting the genetic root cause. The single participant will receive the drug and be monitored f…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 17, 2026 11:07 UTC
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One-of-a-kind drug aims to help rare genetic disorder
Disease control Not yet recruitingThis study tests a personalized medicine called an antisense oligonucleotide (ASO) designed specifically for one person with Schuurs-Hoeijmakers syndrome, a rare genetic condition caused by a specific PACS1 gene mutation. The goal is to see if the drug can improve communication a…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 08, 2026 12:04 UTC