SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Epicrispr Biotechnologies, a clinical-stage company pioneering gene-modulating therapies, today announced that the first patient has been dosed in its global first-in-human clinical trial of EPI-321, an investigational, one-time epigenetic editing therapy for facioscapulohumeral muscular dystrophy (FSHD). FSHD is a progressive genetic disease...
In a number of company updates, Avidity Biosciences announced the FDA’s openness for an accelerated approval for its investigational agent delpacibart braxlosiran (del-brax) as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD). The company has also initiated its phase 3 FORWARD study, a confirmatory, large-scale...
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 three-part Phase 1/2...
On May 8, 2024, Chris and Laura Carrino joined with members of the FSHD Society for a trip to Capitol Hill to meet with Senators, Congressman, and staffers in an effort to advocate for FSHD. With promising clinical trials underway, there is an urgency to introduce...
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...
Read original article and view video content here. Brooklyn Nets' radio announcer Chris Carrino is being honored by the NBA for both his decades of work and the obstacles he has overcome. After more than 20 years as the radio play-by-play voice of the Nets, Carrino has...
This article originally appeared on netsdaily.com. The NBA is honoring Chris Carrino, the play-by-play announcer, with the prestigious NBA Values of the Game Award this year, the Nets and the league reported Tuesday. The award recognizes an individual at an NBA team who exemplifies the values of the...
“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....
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