RNA-Therapien:
Zelltherapie:
Fazirsiran (Takeda) https://clinicaltrials.gov/study/NCT05677971
Stammzellen & Lunge https://clinicaltrials.gov/study/NCT06892236?term=stem%20cell&cond=AATD&rank=1
Wave Life Science WVE-006 https://clinicaltrials.gov/study/NCT06405633?term =wave&cond=Alpha1-antitrypsin%20Deficiency&rank=1
Ersatztherapie:
Kamada Inhalationstherapie https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-kamada-aat-for- inhalation-in-adults-with-alpha-1-antitrypsin-deficiency-and-moderate-to-severe-breathing-issues/
Korro Bio KRRO-110 https://clinicaltrials.gov/study/NCT06677307?cond =Alpha1-antitrypsin%20Deficiency&aggFilters=status:not%20rec&rank=7
Sanofi INBRX-101 https://clinicaltrials.gov/study/NCT05897424?cond=Alpha1-antitrypsin%20Deficiency&term =sanofi&rank=2#study-overview
BioMarin BMN 349 https://clinicaltrials.gov/study/NCT06738017?cond =Alpha-1%20Antitrypsin%20Deficiency%20%5C(AATD%5C)&term=Biomarin&rank=1
Personalisierte Medizin:
Lungentherapien:
Alvelestat (Mereo) https://clinicaltrials.gov/study/NCT03636347?cond =Alpha-1%20Antitrypsin%20Deficiency%20%5C(AATD%5C)&term=mereo&rank=1
EARCO Register https://www.tandfonline.com/doi/full/10.1080/15412555.2024.2393348#abstract
EARCO Studie https://clinicaltrials.gov/study/NCT04180319?term=EARCO&cond=Alpha1- antitrypsin%20Deficiency&rank=1
Gentherapie:
Beam-302 – Erklärung https://clinicaltrials.eu/inn/gr0015/#:~:text=such%20as%20cirrhosis.-,How% 20BEAM%2D302%20Works,genetic%20mutation%20responsible%20for%20AATD
Frühdiagnose:
Krystal Biotech KB408 https://clinicaltrials.gov/study/NCT06049082?cond =Alpha-1%20Antitrypsin%20Deficiency%20%5C(AATD%5C)&term=SERPENTINE-1&rank=1
Neugeborenen-Screening https://clinicaltrials.gov/study/NCT05687474?term=newborn&cond=Alpha1- antitrypsin%20Deficiency&rank=5
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