By J Waters In the late 19th century, two French neurologists described a new form of muscular dystrophy which they named for the specific muscles it affects—the facial (facio-), shoulder (-scapulo-), and upper arm muscles (-humeral). Facioscapulohumeral dystrophy, or FSHD, causes progressive muscle weakness and wasting in...
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 Justin Cohen, PhD Avidity Biosciences recently announced exciting interim results from their randomized, double-blind, placebo-controlled Phase 1/2 FORTITUDE trial in facioscapulohumeral muscular dystrophy (FSHD). Termed delpacibart braxlosiran or del-brax for short, this first-in-class therapy is a muscle targeting antibody bound to a payload that targets...
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...
By Justin Cohen, PhD Kate Therapeutics, a San Diego base biotechnology company has announced funding to advance their muscle and cardiovascular disease portfolio. Among the first diseases to be explored is Facioscapulohumeral muscular dystrophy (FSHD), exciting news for patients like myself and their families who are impacted from this...
By Justin Cohen, PhD Air-travel for wheelchair users come with a unique set of challenges that cause many to forgo the plane altogether. This could all change after the recent Aircraft Interiors Expo in Hamburg, Germany, which unveiled a new airplane seat prototype that could revolutionize air-travel for...
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...
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