Bonnie shares her story of being diagnosed with Alpha-1 Antitrypsin Deficiency and learning to manage the rare disease.
Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition characterised by the misfolding of the alpha-1 antitrypsin (AAT) protein, resulting in its hepatic accumulation and compromised protease ...
NEW YORK – BioMarin has stopped developing the investigational BMN 349 for treating alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the company announced in a regulatory filing this ...
Sanofi (EURONEXT: SAN) announced Wednesday that its investigational therapy, efdoralprin alfa, has received orphan drug ...
Sanofi’s efdoralprin alfa earns orphan designation in the EU for alpha-1 antitrypsin deficiency related emphysemaAdditional orphan designation ...
TSRA-196 is a potential one-time treatment to precisely correct the genetic mutation underlying AATD, with Investigational New Drug filing expected by the end of the year Tessera to receive $150 ...
ROCHESTER MINN. -- Mayo Clinic scientists have discovered that carriers of a genetic defect previously linked to emphysema have a threefold increased risk of developing a type of sporadic colorectal ...
(RTTNews) - Beam Therapeutics Inc. (BEAM) announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a ...
MOLINE, Ill. — November is Alpha-1 Awareness Month, a time dedicated to increasing understanding of Alpha-1 Antitrypsin Deficiency, a rare genetic condition that can cause serious lung and liver ...
Alpha-1 antitrypsin (AAT) deficiency is a genetic disease where your body doesn’t make enough normal functioning AAT. AAT is a protein your liver makes. AAT has many important roles in your body, ...
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