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Drug discovery in focal and segmental glomerulosclerosis.
Authors Pullen N, Fornoni A
Submitted By Alessia Fornoni on 10/25/2016
Status Published
Journal Kidney international
Year 2016
Date Published
Volume : Pages 89 : 1211 - 20
PubMed Reference
Abstract Despite the high medical burden experienced by patients with focal segmental
glomerulosclerosis, the etiology of聽the condition remains largely unknown. Focal
segmental glomerulosclerosis is highly heterogeneous in clinical and聽morphologic
manifestations. While this presents challenges for the development of new
treatments, research investments over the last 2 decades have yielded a surfeit
of potential avenues for therapeutic intervention. The development of many of
those ideas and concepts into new therapies, however, has been very
disappointing. Here, we describe some of the factors that have potentially
contributed to the poor translational performance from this research investment,
including the confidence we ascribe to a target, the conduct of experimental
studies, and the availability of selective reagents to test hypotheses. We will
discuss the significance of genetic and聽systems traits as well as other methods
for reducing bias. We will analyze the limitations of a successful drug
development. We will use specific examples hoping that these will guide a
consensus for investment and drive greater translational quality. We hope that
this substrate will serve to exemplify the tremendous opportunity for
intervention as well as facilitate greater collaborative effort between
industry, academia, and private foundations in promoting appropriate validation
of these targets. Only then will we have achieved our goal for curative
therapies for this devastating disease.


Investigators with authorship
NameInstitution
Alessia FornoniUniversity of Miami - Medical Campus

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