TY - JOUR
T1 - Current Landscape of Treatments for Wolfram Syndrome
AU - Abreu, Damien
AU - Urano, Fumihiko
N1 - Funding Information:
This work was partly supported by the grants from the National Institutes of Health/NIDDK (DK112921, DK113487, DK020579), National Institutes of Health/NCATS (TR002065, TR002345), JDRF (2-SRA-2016-233-S-N) and philanthropic support from the Unravel Wolfram Syndrome Fund, the Silberman Fund, the Stowe Fund, the Snow Foundation, the Eye Hope Foundation, the Ellie White Foundation for the Rare Genetic Disorders, Wolfram Syndrome UK Support Group, French Wolfram Association, and the Feiock Fund to F.U. The authors thank all the members of the Washington University Wolfram Syndrome Study and Research Clinic for their support (https://wolframsyndrome.dom.wustl.edu) and all the participants in the Wolfram syndrome International Registry and Clinical Study, Research Clinic, and Clinical Trials for their time and efforts. F. Urano received research funding from Eli Lilly, Ono Pharmaceutical, and Amarantus BioScience for the development of MANF-based regenerative therapy. He also received chemical compounds from Amylyx Pharmaceuticals, Mitochon Pharmaceuticals, Aetas Pharma, and National Center for Advancing Translational Sciences for the development small molecule-based therapies targeting ER stress.
Funding Information:
This work was partly supported by the grants from the National Institutes of Health /NIDDK ( DK112921 , DK113487 , DK020579 ), National Institutes of Health /NCATS ( TR002065 , TR002345 ), JDRF ( 2-SRA-2016-233-S-N ) and philanthropic support from the Unravel Wolfram Syndrome Fund, the Silberman Fund, the Stowe Fund, the Snow Foundation, the Eye Hope Foundation, the Ellie White Foundation for the Rare Genetic Disorders, Wolfram Syndrome UK Support Group, French Wolfram Association, and the Feiock Fund to F.U. The authors thank all the members of the Washington University Wolfram Syndrome Study and Research Clinic for their support ( https://wolframsyndrome.dom.wustl.edu ) and all the participants in the Wolfram syndrome International Registry and Clinical Study, Research Clinic, and Clinical Trials for their time and efforts.
Funding Information:
F. Urano received research funding from Eli Lilly, Ono Pharmaceutical, and Amarantus BioScience for the development of MANF-based regenerative therapy. He also received chemical compounds from Amylyx Pharmaceuticals, Mitochon Pharmaceuticals, Aetas Pharma, and National Center for Advancing Translational Sciences for the development small molecule-based therapies targeting ER stress.
Publisher Copyright:
© 2019 The Author(s)
PY - 2019/10
Y1 - 2019/10
N2 - Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
AB - Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
KW - WFS1
KW - Wolfram syndrome
KW - endoplasmic reticulum
KW - gene therapy
KW - regenerative therapy
UR - http://www.scopus.com/inward/record.url?scp=85070489460&partnerID=8YFLogxK
U2 - 10.1016/j.tips.2019.07.011
DO - 10.1016/j.tips.2019.07.011
M3 - Short survey
C2 - 31420094
AN - SCOPUS:85070489460
SN - 0165-6147
VL - 40
SP - 711
EP - 714
JO - Trends in Pharmacological Sciences
JF - Trends in Pharmacological Sciences
IS - 10
ER -