Four-day CureDuchenne conference will feature key opinion leaders and notable speakers including Peter Marks, M.D., Ph.D., of U. S. Food & Drug Administration

Newport Beach, Calif. (May 8, 2024) – CureDuchenne,a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, announced its FUTURES National Conference will be held from May 23-26 in Orlando, Florida. FUTURES is a four-day event focused on bringing education, connection and hope to the Duchenne and Becker community. The annual event will take place as a hybrid event, offering attendees a chance to participate both online and in-person, at the JW Marriott Orlando, Grande Lakes.

This year’s event theme is “Embrace our Future,” placing importance on proactive care and treatment options, community support, advocacy and inclusivity and hope for the future. CureDuchenne experts and industry leaders in research, healthcare, and biotechnology will offer insightful and interactive presentations on a variety of relevant topics, including the latest research and therapies for individuals with Duchenne and Becker muscular dystrophy.

Family support will be a focus of the conference, with sessions for newly diagnosed families, single parents, siblings, adults with Duchenne, and those affected by behavioral challenges. CureDuchenne will also share data updates on CureDuchenne Link, its data-integrated biobank, and attendees with Duchenne or Becker muscular dystrophy, as well as genetic carriers, will be able to enroll onsite to support research toward a cure for Duchenne. The four-day conference also features social events for the whole family, including an unforgettable swim with dolphins experience an all-inclusive, fun-filled day at Discovery Cove, made possible through a partnership with SeaWorld and with the support of a generous anonymous donor.

Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER), will deliver the keynote address to kick off the FUTURES National Conference for families. CBER is responsible for regulation and approvals for all cell and gene therapies, including the 2023 accelerated approval of the first gene therapy for 4-5-year-olds in Duchenne. Dr. Marks will share his insights on advancing cell and gene therapies for diseases like Duchenne muscular dystrophy.

“In the rapidly evolving landscape of Duchenne research, the connection between families, researchers, and clinicians has never been more critical. It is essential that families have direct access to the latest advancements and insights from the forefront of Duchenne research. We’re also honored to include Dr. Marks as our keynote speaker, allowing families to hear the latest on approvals directly from the FDA,” said Debra Miller, founder and CEO of CureDuchenne. “At CureDuchenne, we are dedicated to providing a platform for dialogue and understanding that empowers families to navigate this complex terrain. The FUTURES National Conference embodies this mission, bringing together the community to share, learn, and support each other in our collective journey toward a cure for those affected by Duchenne and Becker muscular dystrophy.”

Prior to the public conference, CureDuchenne will convene scientific leaders, executives from pharmaceutical and biotech companies focused on developing therapies for Duchenne and Becker muscular dystrophy, and regulatory leaders for the Meeting of the Minds, a private roundtable discussion on addressing the challenges and opportunities in bringing treatments to individuals who need them. This discussion will include Dr. Marks as well as Peter Stein, M.D., the Director of CDER’s Office of New Drugs (OND), at the U.S. Food & Drug Administration.

The event for families will start on Thursday, May 23 with meetups for newly diagnosed families and adults with Duchenne and a welcome reception. Research sessions throughout the conference will highlight clinical trial design, gene therapy and gene editing, RNA-targeted therapeutics, and muscle preservation and anti-inflammatory treatment approaches. Educational sessions will include topics such as building success at school, navigating adolescence, responding to emergencies, supportive care, physical therapy, and resources for adults with Duchenne and female Duchenne carriers. Translation is available for all sessions, and select sessions are designed specifically for Spanish-speaking families with Duchenne.

Throughout the weekend, CureDuchenne will offer on-site childcare and activities for the whole family, including a gaming lounge for kids and a hangout lounge for adults with Duchenne. For registration information, full agenda and speaker list, please visitcureduchenne.org/futures.

About CureDuchenne

Over twenty years ago, CureDuchenne was created with one goal: to find and fund a cure for Duchenne muscular dystrophy, one of the most common and severe forms of muscular dystrophy. Today, CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne. CureDuchenne’s venture philanthropy approach has advanced transformative treatments for Duchenne muscular dystrophy, including 18 projects that advanced to human clinical trials and multiple projects to overcome the limitations of exon-skipping and gene therapy. In addition, CureDuchenne contributed early funding to the first FDA-approved Duchenne drug, pioneered the first and only Duchenne physical and occupational therapist certification program and created an innovative data-integrated biobank, accelerating research toward a cure.

For more information on how to help raise awareness and funds needed for research, please visithttps://cureduchenne.org,or follow us onFacebook, Instagram, LinkedIn,andTwitter.

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