Facioscapulohumeral muscular dystrophy (FSHD) is one of the most prevalent neuromuscular disorders. The disease is linked to copy number reduction and/or epigenetic alterations of the D4Z4 macrosatellite on chromosome 4q35 and associated with aberrant gain of expression of the transcription factor DUX4, which triggers a pro-apoptotic transcriptional program leading to muscle wasting. As today, no cure or therapeutic option is available to FSHD patients. Given its centrality in FSHD, blocking DUX4 expression with small molecule drugs is an attractive option.
“The progression of FSHD can make it increasingly difficult to pursue work, hobbies, social and family life,” Mark Stone, CEO of the FSHD Society, said. “The REACH trial with losmapimod represents the first real hope for those living with the disease, their families and their caregivers, who have waited so long for a potential treatment to be found.”
EPIC BIO, a biotechnology company developing ultracompact therapies to modulate gene expression in vivo, today announced its launch and Series A financing of $55 million. Epic was founded by Stanley Qi, Ph.D., a renowned bioengineer and named co-inventor on the CRISPR patent held by the University of California. The company’s mission is to develop a new class of genetic medicines that act on the epigenome, a naturally occurring mechanism that determines which genes are expressed and at what levels.
Chip Wilson, founder of the athletic-inspired apparel company Lululemon, has committed $100 million to find a cure for his rare disease, facioscapulohumeral muscular dystrophy type 2.
A team of Florida State University researchers has found that using sophisticated gene-editing techniques, they can essentially turn off or remove part of the genetic code that leads to the disease
Fulcrum Therapeutics has announced positive results with its ReDUX4 trial for losmapimod, providing new hope for the Facioscapulohumeral muscular dystrophy (FSHD) community after decades of unsuccessful clinical trials.
The Chris Carrino Foundation for FSHD is proud to announce funding of $50,000 to the Jones Lab at The University of Nevada, Reno School of Medicine to help develop a larger animal model in pursuit of a treatment for FSHD.
The Chris Carrino Foundation, in conjunction with Friends of FSHD Research, is proud to continue funding for the Dr Suja Janaggathan Lab at The University of Colorado. We have just approved funding of year two of this project, with our portion amounting to $46,344.50.
CRISPR-Cas9, originally discovered in prokaryotic organisms to be part of their immune system, has rapidly developed into a gene editing tool far more accurate than its predecessors. This technology is thus generating a lot of buzz due to its potential to correct genetic diseases. However, what if it could be used to identify factors that can lessen disease severity? These targets could be the source of novel treatments. A recent study out in Science Translational Medicine led by Angela Lek PhD and Louis Kunkel PhD explored this in Facioscapulohumeral Muscular Dystrophy (FSHD), a type of muscular dystrophy for which there is no treatment or cure.
https://youtu.be/LXr1L4NOmos
