Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD

WATCH TIME: 2 minutes

“Of course, we have approved therapies already, but many more are in the clinical pipeline and clinical trials, earlier [in disease] and more advanced. The results coming out—good and promising results, but also the problems that we have to face jointly—give us a much more granular picture of gene therapy as well as the risks and other aspects we need to manage.”

At the 2022 MDA Clinical & Scientific Conference, March 13-16, in Nashville, Tennessee, the opening ceremony featured a presentation of the first annual MDA Legacy Award for Excellence in Clinical Research, which was given to Carsten G. Bönnemann, MD, senior investigator, Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH). Bönnemann has long conducted research in the field of neuromuscular disorders and was awarded the honor by MDA National Ambassador Amy Shinneman, a patient with Bethlem myopathy.

While on-site in Nashville, Bönnemann sat down with NeurologyLive^® to discuss what the award meant to him and his team at the NIH, noting that it marked a culmination of sorts of a long-held goal of their research efforts: to integrate patient care with genetic knowledge to produce genetic therapies. This goal has also been reflected by the progress made by the neuromuscular disease field, which has seen rapid advances in therapeutics for a variety of patient populations, including amyotrophic lateral sclerosis, spinal muscular atrophy, and Duchenne muscular dystrophy, among others.

Bönnemann also shared his perspective on this progress that’s been made, particularly in the last year, with genetic therapies and testing efforts. He put much emphasis on the headway gained in genetic medicine, as a few notable genetic approaches have begun to make their way to critical phases of development and clinical trial assessment. The results of this work to this point and what will come in the next several years, Bönnemann noted, have been and will be essential in providing a clearer picture of therapeutic optimization for patients with neuromuscular disease.

For more coverage of MDA 2022, click here.