CureDuchenne to Host Webinars After FDA Expands ELEVIDYS Label

CureDuchenne, a prominent global nonprofit focused on curing Duchenne muscular dystrophy, has announced upcoming webinars in English and Spanish for the Duchenne community. This follows the U.S. Food and Drug Administration (FDA) approving an expanded label indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) from Sarepta Therapeutics. The approval now includes individuals with a confirmed DMD gene mutation who are at least four years old.

The webinars will be overseen by Dr. Michael Kelly, Chief Scientific Officer at CureDuchenne, and will feature Dr. Diana Castro, a board-certified neurologist and prominent neuromuscular physician. Dr. Castro will share the latest insights about the gene therapy treatment and her experiences as a physician treating patients with Duchenne muscular dystrophy. Additionally, a representative from Sarepta Therapeutics will attend as a guest for a Q&A session.

The English session is scheduled for Tuesday, June 25, 2024, at 3:00 PM PST, followed by the Spanish session at 4:30 PM PST on the same day.

CureDuchenne was established over two decades ago with a singular mission: to find and fund a cure for Duchenne muscular dystrophy, which is one of the most prevalent and severe forms of this genetic disorder. Over the years, CureDuchenne has earned a reputation as a global leader in research, patient care, and innovative solutions aimed at improving and prolonging the lives of those affected by Duchenne muscular dystrophy.

The organization employs a venture philanthropy approach, which has significantly contributed to advancing transformative treatments for the disease. This approach has led to 18 projects moving forward to human clinical trials and several initiatives designed to overcome the limitations of exon-skipping and gene therapy. Furthermore, CureDuchenne provided early funding for the first FDA-approved drug for Duchenne and pioneered the only certification program for Duchenne physical and occupational therapists. They also developed a cutting-edge data-integrated biobank to speed up research efforts toward finding a cure.

CureDuchenne has been instrumental in various innovative projects and initiatives aimed at combating Duchenne muscular dystrophy. Their work includes contributing to the early stages of clinical trials for new treatments, overcoming challenges associated with exon-skipping and gene therapy, and developing a biobank that integrates data to accelerate research. The nonprofit's efforts also extend to creating certification programs for therapists, ensuring that patients receive specialized care.

CureDuchenne continues to play a crucial role in the global fight against Duchenne muscular dystrophy by funding research, fostering innovation, and improving patient care. Their dedication to these efforts has not only advanced scientific understanding and treatment options but also provided hope to many individuals and families affected by this debilitating disease.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!