Tag: FSHD

“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.

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.