First nonsteroidal treatment approved for patients six years of age and older with DMD regardless of genetic mutation now available in the U.S.  ITF also announces the launch of ITF […] The post ITF Therapeutics LLC Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Patients With Duchenne Muscular Dystrophy  appeared first on CureDuchenne ..read more

READ THE FULL PRESS RELEASE HERE Introduction The journey towards finding effective treatments for Duchenne Muscular Dystrophy (DMD) has been marked by significant milestones. However, recent findings indicate that we […] The post Expanding Duchenne Clinical Trial Criteria: A Crucial Step Forward appeared first on CureDuchenne ..read more

We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new initiative represents a significant step forward in enhancing support and treatment options for rare disease patients, particularly those living with Duchenne and Becker muscular dystrophy. Streamlining Treatment Development The Rare Disease Innovation Hub is a collaborative effort between the FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER ..read more

As World Duchenne Day (WDD) approaches on September 7, 2024, we at CureDuchenne invite you to join us in raising awareness and showing support for our Duchenne community. This year, we have prepared a special social media graphics package for you. How to Support World Duchenne Day Post on Social Media Take a Photo Use the Template: Enhance your post using the template provided in this link Change Your Profile Picture: Download and customize your profile picture here. Customization Option: If you prefer, send your photo to marketing@cureduchenne.org, and we will customize it for you and send i ..read more

 Event Has Raised More Than $5.8 Million Over the Last 12 Years to Drive a Cure for Duchenne Muscular Dystrophy  NEWPORT BEACH, Calif., (July 12, 2024) — CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, will partner once again with NHL All-Star and retired Anaheim Ducks captain Ryan Getzlaf for the 13th annual Getzlaf Golf Shootout at Monarch Beach Golf Links on September 13-14, 2024. Hosted by Ryan Getzlaf and his wife Paige, this annual event brings together professional athletes, celebrities, and community leaders for ..read more

Dyne Therapeutics shares with the community recent efficacy and safety data from the ongoing DELIVER trial of DYNE-251 in Duchenne. Watch HERE The post Dyne Therapeutics of DYNE-251 in Duchenne appeared first on CureDuchenne ..read more

CureDuchenne Webinar | ITF THERAPEUTICS Patient Community Update: An overview of DUVYZAT Recorded on June 26, 2024 Watch HERE The post ITF THERAPEUTICS| DUVYZAT Update appeared first on CureDuchenne ..read more

Watch HERE The post Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys appeared first on CureDuchenne ..read more

This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. This therapy, while not a cure, aims to improve the quality of life for patients. The treatment involves a single dose, but patients must undergo extensive pre-treatment screening to help mitigate the chances severe immune reactions. Key points include understanding the genetic mutation specifics, managing potential side effects, and adhering to post-treatment protocols. The process includes g ..read more

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