friends of FSH Research

Tagline

Search News Items

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

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.
Harper's lab had previously designed two miRNAs, labeled mi1155 and mi405, and.had performed a low dosage test on mouse muscle. This paper describes what happened when the dosage … http://fshfriends.com/blog/pre-clinical-safety-and-target-studies-support-translation-aav-mediated-rnai-therapy-fshd/

Progress Report: Utilization of cell-free DNA as a biomarker

Initial progress report provided by Premi Haynes PhD.
by Premi Haynes See grant: Utilization of cell-free DNA (cfDNA) as a sensitive biomarker for clinical trial end point for individuals with FSHD Cell free DNA (cfDNA) are pieces… http://fshfriends.com/blog/progress-report-utilization-cell-free-dna-biomarker/

Progress Report: Clinical Study using MRI to measure immune response

Dr. Wang provides a progress report on his clinical study.
I think that this progress report exemplifies the trials and tribulations that all researchers go through in putting together a human clinical study. A lot of regulatory bodies, e… http://fshfriends.com/blog/progress-report-clinical-study-using-mri-measure-immune-response/

Grant Awarded to Develop Outcome Measure

A proposal to study the histopathological features of muscle in a large biobank from FSHD patients and their first degree family members.
See grant: Histochemical and morphometric analysis of FSHD biceps for Kathryn R Wagner MD PhD. This is a proposal to study the histopathological features of muscle in a large bi… http://fshfriends.com/blog/grant-awarded-develop-outcome-measure/

aTyr announces initial results in FSHD trial

aTyr pharma has encouraging news regarding a possible treatment for FSHD using a novel protein-based biologic drug.
aTyr recently released a press statement reporting their initial results from their FSHD study.  The treatment was developed for use in myopathies with an immune component.  This … http://fshfriends.com/blog/atyr-announces-initial-results-fshd-trial/

Participate in FSHD research: UW/Children's research study

FSHD clinical research project needs patients to complete study
Researchers at the University of Washington and Seattle Children's Hospital are seeking volunteers for a study on FSHD. Within the next few years researchers are expecting that… http://fshfriends.com/blog/participate-fshd-research-uwchildrens-research-study/

Participate in FSHD Research

Researchers at the University of Washington and Seattle Children's Hospital are seeking volunteers for a study on FSHD.
Researchers at the University of Washington and Seattle Children's Hospital are seeking volunteers for a study on FSHD. Within the next few years researchers are expecting th… http://fshfriends.com/blog/participate-fshd-research/