TY - JOUR
T1 - Alport Syndrome Therapeutics
T2 - Ready for Prime-Time Players
AU - Omachi, Kohei
AU - Miner, Jeffrey H.
N1 - Funding Information:
The authors are supported by grants from the Japan Society for the Promotion of Science postdoctoral fellowships for research abroad (to K.O.), the National Institutes of Health (NIH) ( R01DK078314 and R01DK058366 ) to J.H.M., and the Children's Discovery Institute of Washington University and St. Louis Children's Hospital (to J.H.M.). The authors thank Rachel Lennon for helpful comments on the manuscript and apologize for being unable to cite much of the primary literature due to space limitations.
Funding Information:
J.H.M. has received grant support from Reneo Pharmaceuticals and Reata Pharmaceuticals and has served as a consultant to Regulus and Retrophin.
Funding Information:
The authors are supported by grants from the Japan Society for the Promotion of Science postdoctoral fellowships for research abroad (to K.O.), the National Institutes of Health (NIH) (R01DK078314 and R01DK058366) to J.H.M. and the Children's Discovery Institute of Washington University and St. Louis Children's Hospital (to J.H.M.). The authors thank Rachel Lennon for helpful comments on the manuscript and apologize for being unable to cite much of the primary literature due to space limitations. J.H.M. has received grant support from Reneo Pharmaceuticals and Reata Pharmaceuticals and has served as a consultant to Regulus and Retrophin.
Publisher Copyright:
© 2019 Elsevier Ltd
PY - 2019/11
Y1 - 2019/11
N2 - Alport syndrome (AS), a rare disease of basement membrane type IV collagen, impacts the kidneys, ears, and eyes. In severe cases, kidney failure occurs during adolescence or early adulthood, so most research has focused on remedies for kidney dysfunction. Planned and ongoing clinical studies using targets and therapeutic approaches discussed herein provide new hope for AS patients. The outcomes of these trials could suggest new treatments for more common forms of chronic kidney disease (CKD), demonstrating the importance of focusing on treatments for rare diseases.
AB - Alport syndrome (AS), a rare disease of basement membrane type IV collagen, impacts the kidneys, ears, and eyes. In severe cases, kidney failure occurs during adolescence or early adulthood, so most research has focused on remedies for kidney dysfunction. Planned and ongoing clinical studies using targets and therapeutic approaches discussed herein provide new hope for AS patients. The outcomes of these trials could suggest new treatments for more common forms of chronic kidney disease (CKD), demonstrating the importance of focusing on treatments for rare diseases.
KW - chronic kidney disease
KW - collagen IV
KW - glomerulus
KW - hematuria
UR - http://www.scopus.com/inward/record.url?scp=85071079923&partnerID=8YFLogxK
U2 - 10.1016/j.tips.2019.07.012
DO - 10.1016/j.tips.2019.07.012
M3 - Short survey
C2 - 31455555
AN - SCOPUS:85071079923
SN - 0165-6147
VL - 40
SP - 803
EP - 806
JO - Trends in Pharmacological Sciences
JF - Trends in Pharmacological Sciences
IS - 11
ER -