ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Experimental drug for rare liver disease shows promise but study halted early
Disease control TerminatedThis study tested a new drug called belcesiran in people with a rare genetic condition (Alpha-1 antitrypsin deficiency) that causes liver damage. The goal was to see if the drug is safe and can reduce harmful protein buildup in the liver. The study was stopped early, so results a…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company • Aim: Disease control
Last updated May 26, 2026 05:08 UTC
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Early-Stage trial for rare lung disease halted before completion
Disease control TerminatedThis early-stage study tested a new drug called ARC-AAT in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can damage the lungs and liver. The main goal was to check the drug's safety and how it affects AAT protein levels in the …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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New drug shows promise for rare lung and liver disease
Disease control TerminatedThis study tested an experimental drug called VX-864 in 14 adults with Alpha-1 Antitrypsin Deficiency who have the PiZZ genetic type. The goal was to see if the drug could raise levels of a protective protein in the blood and reduce harmful protein clumps in the liver. The study …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 22, 2026 14:02 UTC