N-lorem Foundation
Clinical trials sponsored by N-lorem Foundation, explained in plain language.
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One-of-a-kind drug targets rare ALS gene in first human test
Disease control Not yet recruitingThis study tests a personalized medicine designed for one person with ALS caused by a specific CHCHD10 gene mutation. The drug is an antisense oligonucleotide (ASO) that aims to slow disease progression. The single participant will be monitored for changes in muscle strength, bre…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 22, 2026 14:05 UTC
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Custom Gene-Targeting drug tested for rare developmental disorder
Disease control Not yet recruitingThis study tests a custom-made drug designed for one person with Schuurs-Hoeijmakers syndrome, a rare genetic condition that affects development. The drug, called an antisense oligonucleotide, aims to correct the effects of a specific gene mutation. The trial will measure changes…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 22, 2026 13:55 UTC