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Researchers at Mayo Clinic's Center for Individualized Medicine have discovered a rare genetic variant that can directly ...
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Alpha-1 antitrypsin deficiency: Study reveals why some patients with the hereditary disease remain healthyStudying a monogenic condition like Alpha-1 antitrypsin deficiency ... Rosenberger et al, Deep Visual Proteomics maps ...
The first three approved RNAi drugs — all discovered and developed at Alnylam — were for rare, monogenic diseases with targets expressed in the liver. As confidence in the technology grew ...
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This integrated trio of care wasn’t always available to patients with liver disease, says Courtney Sherman, MD, hepatologist and associate professor at UC San Francisco. HALT founders Courtney Sherman ...
Opti-HEP models are now routinely used for the identification of novel therapies against rare monogenic liver diseases including alpha-1-antitrypsin deficiency (A1ATD) and Wilson’s disease.
Fatty liver disease, medically called metabolic dysfunction-associated steatotic liver disease (MASLD), affects millions globally. Around one in four adults worldwide has this hidden condition.
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