Articles: disease.
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There is a paucity of data on treatment of osteoporosis in patients with advanced chronic kidney disease (CKD). ⋯ U.S. Food and Drug Administration.
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Danon disease is a rare, X-linked, monogenic cardiomyopathy caused by mutations in the lysosomal-associated membrane 2 gene (LAMP2), which encodes the LAMP2 protein. In male patients, the predominant phenotype is progressive cardiac hypertrophy, cardiac dysfunction, and early death. There are no directed therapies for the disease. ⋯ A single infusion of RP-A501 appeared to be safe and was associated with cardiac LAMP2 expression and evidence of clinical improvement over a period of 24 to 54 months. (Funded by Rocket Pharmaceuticals; ClinicalTrials.gov number, NCT03882437.).
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Guidelines currently recommend annual screening for albuminuria only among persons with diabetes mellitus (DM). There is no guidance about albuminuria screening in those with other important risk factors for chronic kidney disease (CKD), such as hypertension and/or family history of kidney disease. We sought to create a risk score that predicts the likelihood of albuminuria in adults without diabetes to prompt earlier detection and management of CKD. ⋯ These results suggest that it may be helpful to use a risk score framework for albuminuria screening in people without DM to encourage earlier detection and management of CKD. Longitudinal studies are warranted to confirm this approach along with evaluation of its cost effectiveness.