Jun 15, 2018
Grant recipient Premi Haynes discusses her work and interest in FSHD.
Jun 1, 2018
Genea Biocells announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905,
May 30, 2018
Exciting new research being funded both by your investments and through our co-funding partners.
May 25, 2018
As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period of 18 months.
May 25, 2018
Identification of several types of small noncoding RNAs including known microRNAs that are differentially expressed in FSHD2 muscle cells compared to control.
Apr 15, 2018
Recent grant recipient Dr. Anna Pakula discusses her work.
Apr 10, 2018
Text of Lukas Kostka speech given at January Auction.
Mar 19, 2018
ENMC 25th anniversary summit - The Patient Role in Shared Decision Making.
Feb 1, 2018
Slide decks for Dr. Tapscott & Dr. Wang from the 27-Jan presentations.
Jan 17, 2018
A publication from Scott Harper's lab reporting on the use of an AAV vector to test the safety of two miRNAs in silencing DUX4.
Jan 1, 2018
Yi-Wen Chen DMV PhD provides an update on investigating 3rd generation oligonucleotides.
Dec 31, 2017
Initial progress update provided by Premi Haynes PhD.
Dec 16, 2017
Follow-on publication for previously funded grant.
Dec 15, 2017
Jocelyn Eidahl, Post-Doctoral Scientist at Nationwide Children’s Hospital provides a progress update.
Dec 14, 2017
Final progress update from Dr. Wagner.
Nov 28, 2017
A discussion on recent research advances at the Hyatt prior to the Annual Auction.
Nov 27, 2017
Have your Giving Tuesday donations matched through facebook and The Gates Foundation.
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