WOLFRAM SYNDROME
Clinical trials for WOLFRAM SYNDROME explained in plain language.
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Hope for rare wolfram syndrome: drug aims to slow diabetes progression
Disease control OngoingThis study tests an experimental drug called AMX0035 in 12 adults with Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss. The goal is to see if the drug can help preserve the pancreas's ability to make insulin and to check its safety over up to 4 year…
Matched conditions: WOLFRAM SYNDROME
Phase: PHASE2 • Sponsor: Amylyx Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Gene study aims to solve wolfram syndrome mystery
Knowledge-focused OngoingThis study looks at 45 people with certain changes in the WFS1 gene to figure out if they have a milder, dominant form of wolfram-like syndrome instead of the more severe recessive form. Researchers will use eye scans (OCT) to see if the gene changes are linked to specific eye pr…
Matched conditions: WOLFRAM SYNDROME
Sponsor: Hôpital Necker-Enfants Malades • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:03 UTC