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Progress Update: Clinical Study using MRI to measure immune response
Nov 14, 2018
Update from Dr. Wang on his clinical study.
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Progress Update: CTRN International Sites
Nov 5, 2018
Dr. Tawil provides a brief update on the two CTRN European sites funded.
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Fall RFP 2018 Deadline
Oct 5, 2018
Our latest Request For Proposals is now open for submissions.
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TACT: fully Approaching Drug Development
Aug 31, 2018
Fabiola Bertinotti writes about her week in Vienna as a member of the TREAT-NMD Advisory Committee for Therapeutics (TACT).
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Ask the Expert with Dr. Rabi Tawil
Aug 1, 2018
Dr. Tawil discusses his historical relationship with FSHD and the future of potential therapies.
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Progress Update: A cell culture model of innate immunity for FSHD
Jul 31, 2018
Charles Emerson provides the final update on his recent study.
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Progress Update: Defining the role of post-translational modification on FSHD protein DUX4
Jul 17, 2018
Jocelyn Eidahl, Post-Doctoral Scientist at Nationwide Children’s Hospital provides a progress update.
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FSHD Research Funding Overview 2017
Jul 11, 2018
Reporting on worldwide funding of FSHD research.
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Progress Update: 3rd Gen Oligonucleotide Targeting DUX4 in Mouse Model
Jul 11, 2018
Yi-Wen Chen DMV PhD provides an update on investigating 3rd generation oligonucleotides.
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Muscle Stem Cells from Teratomas
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.
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Progress Update: Optimizing the Utility of the National Registry for FSHD Research and Trial Recruitment
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.
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Where We Are
Jun 25, 2018
Fabiola Bertinotti provides an update on Jaya, who spoke at the 2018 Annual Auction.
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Strength, Responsibility and Progress
Jun 20, 2018
Dave Lukas describes his transformation into an advocate for those with FSHD and participation in clinical trials.
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2018 International Research Congress and Research Planning Meeting
Jun 18, 2018
Summary of progress and presentations at the June 8-9 conference in Las Vegas.
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Ask the Expert with Dr. Premi Haynes
Jun 15, 2018
Grant recipient Premi Haynes discusses her work and interest in FSHD.
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World FSHD Day
Jun 15, 2018
World FSHD Awareness Day.
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Genea Biocells Announces FDA Orphan Drug Designation for GBC0905 for the Treatment of 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,
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Five New Grants Awarded
May 30, 2018
Exciting new research being funded both by your investments and through our co-funding partners.
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Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
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.
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Publication: Small noncoding RNAs in FSHD2 muscle cells reveal both DUX4- and SMCHD1-specific signatures
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.