
FDA Approves Duvyzat (givinostat) for Duchenne Treatment
FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy Treatment.
FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy Treatment.
PTC Therapeutics Provides Key Regulatory Updates
This pre-recorded webinar delves into the world of travel with special guests, Marissa Penrod and Adam Malone. Not only is Marissa an expert on traveling with Duchenne, but Adam is a travel advisor who also happens to live with the condition. We are also be joined by Alan Chaulet, who is the Vice President of All Wheels Up which is a non-profit that is working to increase awareness for safer and more dignified accessible travel. Together, they provide invaluable insights, tips, and stories that will leave you feeling inspired and empowered to embark on your very own adventure.
Here’s What You Missed at the MDA Clinical and Scientific Conference 2024
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
AUSTIN, Texas., February 28, 2024 – CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, and the Revell family of Austin will host the 15th annual fundraiser gala, Champions to CureDuchenne: Disco for Duchenne, on April 6, 2024. Austin’s own “Saturday Night Fever” will be filled with glittering lights and pulsating beats, bringing hundreds of philanthropists, businesspeople, and local community members together under one roof at the University of Texas Golf Club to disco for a purpose. Emmy Award-winning weathercaster Jim Spencer will emcee the event, featuring a vibrant disco dance party, extraordinary auctions, delightful drinks, and a tantalizing culinary experience. All proceeds from the event benefit CureDuchenne as it accelerates scientific research and pioneers educational programs to care for families affected by Duchenne — a progressive, fatal disease affecting 300,000 worldwide.
Dyne Therapeutics will present initial clinical data, first released on January 3rd, 2024, from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
The FDA will make a decision whether or not to grant full approval of Sarepta’s gene therapy, Elevidys, by June 21,2024.
Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone fractures.