#
Jul 11, 2018
Reporting on worldwide funding of FSHD research.
#
Jul 11, 2018
Yi-Wen Chen DMV PhD provides an update on investigating 3rd generation oligonucleotides.
#
Jul 10, 2018
Michael Kyba's lab shows that functional skeletal muscle stem cells can be produced from mouse pluripotent stem cells without genetic modification through teratoma formation. As few as 40,000 teratoma-derived cells can regenerate 80% of total muscle volume, improve force generation, and mature into functional muscle stem cells in vivo.
#
Jul 5, 2018
Dr. Tawil provides an update on year two of his project to get genetic testing performed for those in the registry who do not have it, and thus create a larger viable pool for upcoming therapeutic trials.
#
Jun 25, 2018
Fabiola Bertinotti provides an update on Jaya, who spoke at the 2018 Annual Auction.
#
Jun 20, 2018
Dave Lukas describes his transformation into an advocate for those with FSHD and participation in clinical trials.
#
Jun 18, 2018
Summary of progress and presentations at the June 8-9 conference in Las Vegas.
#
Jun 15, 2018
Grant recipient Premi Haynes discusses her work and interest in FSHD.
#
Jun 15, 2018
World FSHD Awareness Day.
#
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 7, 2018
Spring 2018 Request for Research Proposals.
#
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 4, 2018
Annual Auction for FSHD. NEW RENTON LOCATION.