Everolimus in Patients with Autosomal Dominant Polycystic Kidney Disease
Author(s) -
Gerd Walz,
Klemens Budde,
Marwan Mannaa,
Jens Nürnberger,
Christoph Wanner,
Claudia Sommerer,
Ulrich Kunzendorf,
Bernhard Banas,
Walter H. Hörl,
Nicholas Obermüller,
Wolfgang Arns,
Hermann Pavenstädt,
Jens Gaedeke,
Martin Büchert,
Christoph May,
Harald Gschaidmeier,
Stefan Krämer,
KaiUwe Eckardt
Publication year - 2010
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1003491
Subject(s) - medicine , autosomal dominant polycystic kidney disease , everolimus , polycystic kidney disease , cyst , disease , pi3k/akt/mtor pathway , discovery and development of mtor inhibitors , renal replacement therapy , kidney , pathology , bioinformatics , genetics , biology , signal transduction
Autosomal dominant polycystic kidney disease (ADPKD) is a slowly progressive hereditary disorder that usually leads to end-stage renal disease. Although the underlying gene mutations were identified several years ago, efficacious therapy to curtail cyst growth and prevent renal failure is not available. Experimental and observational studies suggest that the mammalian target of rapamycin (mTOR) pathway plays a critical role in cyst growth.
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