Healx identifies existing drugs that could treat rare diseases, offering a cost effective way to improve patient quality of life
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The Polycystic Kidney Disease Conference
Launched in 2002, this FASEB Science Research Conference (SRC) focuses on the inherited renal cystic diseases including the autosomal dominant (ADPKD) and recessive (ARPKD) forms of polycystic kidney disease (PKD), the overlapping autosomal dominant polycystic liver diseases (ADPLD), and many syndromic forms of PKD, such as ciliopathies.
Identiﬁcation of genes mutated in many of these disorders has improved the understanding of their pathophysiology, made the development of orthologous animal models possible, and facilitated the identiﬁcation of therapeutic targets and the implementation of preclinical and clinical trials.