The Science of Healing: Alpha-1 Antitrypsin Deficiency
This is an interesting video about one professional’s long term research into liver damage caused by Alpha-1 Antitrypsin Deficiency, and the hopeful development of a treatment which aims to limit the damage – with early results showing it may even allow for the damage to be reversed.
While a phase 3 study is recruiting in the US for this drug (Fazirsiran), its worth noting that Dicerna are recruiting for a phase 2 study for their drug (belcesiran – which also uses the principle of RNA interference – RNAi) at St Vincent’s in Melbourne.
More information on the Melbourne trial is below:
Clinicaltrials.gov – NCT04764448
Contact Sue Brenton, ESTRELLA Study Coordinator, St. Vincent’s.
Hospital: St Vincent”s Hospital Melbourne
Principal Investigator: Dr Matthew Conron
ESTRELLA Study Coordinator and site contact: Sue Brenton