Read original article here. by Margarida Maia, PhD | June 20, 2024 AOC 1020, an investigational treatment by Avidity Biosciences that’s now called delpacibart braxlosiran, or del-brax, helped adults with facioscapulohumeral muscular dystrophy (FSHD) grow stronger muscles and extend their arms farther than they could four months earlier. These are early data from FORTITUDE (NCT05747924), a...
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...
“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,...
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...
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...
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