CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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New cystinosis drug shows promise in early trial
Disease control OngoingThis study tests a new oral solution called NPI-001 in people with cystinosis, a rare disease that causes cystine buildup in cells. Researchers want to see if NPI-001 is safe and works better than the current drug cysteamine. About 12 participants aged 10 and older will stop thei…
Matched conditions: CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: CYSTINOSIS
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 01:00 UTC