PILLARS OF RESEARCH
In a short period of time, the Foundation has successfully generated 51 medical research grants across 10 countries, funding all types of research to help drive discoveries that may lead to effective treatments and an ultimate cure for people living with FSHD. With clinical trial readiness around the corner we need your help to fast track treatments and increase the quality of life for those living with FSHD
A CURE FOR FSHD
Research projects that are focused on developing treatments to treat the underlying cause of FSHD progression.
MUSCLE WELLNESS
Research projects that are focused on developing treatments to improve musculoskeletal health, strength and well being.
MUSCLE TECHNOLOGY
Research projects that are focused on developing treatments to improve musculoskeletal health, strength and well being.
Grant 2
Derivation of human embryonic stem cells to aid medical research in FSHD
Grant 3
Biomarkers in FSHD, a metabolome study in blood, urine and muscle
Grant 4
Comparing the DnaselHypersensitive Chromatin Landscape at 4q35 of FSHD and Control Cells
Grant 5
Defining the mechanism controlling musclespecific gene expression in FSHD
Grant 6
Deciphering the longdistance interactions of the D4Z4 array in control and FSHD cells
Grant 7
Molecular Genetic Basis of Facio Scapulo Humeral Dystrophy
Grant 8
Dysregulated Pathways in FSHD: Recreating the FSHD Phenotype
Grant 10
Study of DUX4 and DUX4c gene expression in human embryonic stem cells
Grant 11
The development of an antiDUX4 therapeutic based on chemical inhibitors of DUX4
Grant 12
Culture and Expansion of DUX4 in Human Embryonic Stem Cells Carrying FSHD
Grant 13
Bill Moss AO Fellowship for Dr Leslie Caron
Grant 14
Tissue-specific silencing of the Planar cell polarity gene FAT1 as a causal mechanism for FSHD
Grant 15
DUX4 inhibition as a therapeutic strategy for FSHD
Grant 16
Recreating the human chromosomal genetic defect responsible for FSHD in a mouse model.
Grant 17
Evaluation of antisense strategies to suppress DUX4 expression in FSHD
Grant 18
Study on the clinical features, expression profiling, and quality of life of infantile onset FSHD
Grant 19
FSHD drug discovery based on chemical inhibitors of DUX4
Grant 20
Identification of drugs for the normalization of aberrant FSHD candidate gene expression
Grant 21
Drugtargeting of myoblast fusion as a treatment for FSHD
Grant 22
Increasing SMCHD1 Levels as a Therapy for FSHD1 & FSHD2
Grant 23
Clinical Study of Bone Health in FSHD
Grant 24
Generation of Drosophila-Based Biomedical Models of FSHD
Grant 25
Enhancing BMP signaling to treat FSHD
Grant 26
Functional study of a novel candidate gene for FSH (LRIF)
Grant 27
Preclinical Studies of Fisetin and VBP15 in Facioscapulohumeral Muscular Dystrophy
Grant 28
Application of novel isoflavones in an FSHD hESC model system
Grant 29
Training Agreement to Netherlands
Grant 30
HDL based therapy is a potential treatment for FSHD
Grant 31
AO Transporter
Grant 32
A multicenter natural history and biomarkers study of infantile onset FSHD
Grant 33
Facio Therapies Biotech Investment Grant
Grant 34
Targeting DUX4 using gene-silencing oligonucleotides in FSHD models
Grant 35
FSHD Consensus
Grant 37
The next wave of Whole Genome Sequencing-based FSHD diagnostics, and clinical measures of progress
Grant 38
Small molecule inhibitors of DUX4 as FSHD therapeutics
Grant 39
High throughput chemical screens for activators of SMCHD1, as potential therapeutics for FSHD
Grant 40
Living life with FSHD: who is affected and how.
Grant 41
Characterisation of DUX 4 protein – protein interactions in FSHD cell lines and tissue biopsies by cross-linking and mass-spectrometry
Grant 43
Testing novel therapeutic strategies to combat the metabolic disturbances underlying the muscle pathology of FSHD
Grant 44
Physical function outcome measures in paediatric FSHD
Grant 45
Crystal Structure of DUX4 protein domains
Grant 47
Pre-clinical testing for FSHD CRISPR-inhibition therapy
Grant 1
Investigation into the role of FHL1, Calcineurin and NFAT in reducing muscle wasting in FSHD
Grant 9
Investigation of the role of FHL1 as a novel therapeutic target to reduce muscle wasting and promote muscle regeneration in the frg1 mouse model of FSHD
Grant 36
Effect of creatine monophosphate on strength and muscle mass in children with FSHD
Grant 46
Developing a systemic myostatin-inhibiting gene therapy approach to improve muscle weakness in a new FSHD mouse model
Grant 48
Muscle-in-a-dish, development of an in vitro platform of human skeletal muscle