N-lorem Foundation
Clinical trials sponsored by N-lorem Foundation, explained in plain language.
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One-Patient trial aims to treat Ultra-Rare genetic disorder
Disease control Not yet recruitingThis study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with Schuurs-Hoeijmakers syndrome, a rare genetic condition. The drug aims to correct a specific genetic mutation to improve communication and motor skills. Only one participant …
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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Custom-Made genetic drug tested in single ALS patient
Disease control Not yet recruitingThis study tests a custom-made drug designed for one person with ALS caused by a specific gene mutation (CHCHD10). The drug aims to slow disease progression by targeting the genetic root cause. The participant will be closely monitored for changes in muscle strength, breathing, a…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC