STRENGTHENING the global effort to CURE neuromuscular disease

News Express

Jerry R. Mendell, MD, Receives 2023 Best Abstract Award

Aug 18, 2023, 15:54 PM by Allie Happel
Jerry R. Mendell, MD, has been awarded the 2023 Best Abstract Award for his abstract, “Long-Term Safety and Efficacy in Patients With Duchenne Muscular Dystrophy 4 Years Post-Treatment With Delandistrogene Moxeparvovec in a Phase 1/2a Study.”

Jerry R. Mendell, MD, has been awarded the 2023 Best Abstract Award for his abstract, “Long-Term Safety and Efficacy in Patients With Duchenne Muscular Dystrophy 4 Years Post-Treatment With Delandistrogene Moxeparvovec in a Phase 1/2a Study.”

His winning abstract reports the outcomes of a long-term, open-label, Phase 1/2a study (Study 101; SRP-9001-101; NCT03375164) evaluating the safety and functional outcomes 4 years post-treatment with delandistrogene moxeparvovec. Delandistrogene moxeparvovec (SRP-9001) is an investigational rAAV vector-based gene therapy, designed to compensate for missing dystrophin in DMD by delivering a transgene encoding SRP-9001 dystrophin, an engineered dystrophin protein retaining key functional domains of the wild-type protein.

Dr. Mendell concluded that delandistrogene moxeparvovec was well tolerated 4 years post-treatment. Functional assessments demonstrated long-term sustained stabilization of motor function that was clinically meaningful, at ages where functional decline would be expected based on natural history.

This study helped support the agent’s biologics license application, and in June 2023, SRP-9001 was granted accelerated approval by the FDA for individuals with DMD ages 4-5. Emphasizing the transformative potential of this gene therapy research, Dr. Mendell said, “This accelerated approval is extremely important and really advances the field. It’s the first time we’ll have systemic gene therapy in the field for DMD.”

Dr. Mendell is a leading authority in NM disease research, and his groundbreaking research focuses on the development of gene therapy for DMD. Dr. Mendell is a neurologist at Nationwide Children’s Hospital. He has been the director of the Center for Gene Therapy at The Research Institute of Nationwide Children’s Hospital; director of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center; co-director of the Muscular Dystrophy Association Clinic; and professor of pediatrics, neurology, pathology, physiology, and cell biology at The Ohio State University College of Medicine.

Dr. Mendell also expressed his gratitude and appreciation to AANEM for this recognition, and acknowledged that this accolade serves as a significant encouragement for his team.

frase

Donate Today to Support Research and Researchers' Education

Every dollar raised is a dollar used to support researchers finding a cure. Administrative and staff assistance is provided by AANEM.

Donate Now

 

Apply for Research Grants & Awards

Upcoming Events

Thank You For Your Support

Together we are helping researchers find treatments and cures for tomorrow and improving the lives of patients with neuromuscular diseases.

Donate