Jeremy M. Shefner, MD, PhD, Receives 2026 AANEM Distinguished Researcher Award

Jeremy M. Shefner, MD, PhD, has built a career in NM medicine around a dual commitment: advancing scientific understanding and improving the lives of people living with amyotrophic lateral sclerosis (ALS). As the recipient of the AANEM Distinguished Researcher Award, his work reflects the impact that rigorous research, collaboration, and clinical dedication can have on a rapidly evolving field.

Dr. Shefner's path into NM medicine began with a foundation in electrophysiology during his graduate training, which led him toward clinical neurophysiology. However, it was his early clinical experiences that solidified his direction. "In my resident clinic I encountered several people with ALS," he said. "I found the people tremendously compelling, and the need for better treatments profound." By the end of his training, he had committed himself to both patient care and research aimed at improving treatment options.

Throughout his career, Dr. Shefner has focused on addressing gaps in both knowledge and care. Early on, his research centered on using neurophysiological techniques to improve diagnosis and disease monitoring, as well as adapting these approaches for animal studies of new treatments. As the field advanced, so did his work. "More recently, I have concentrated on how innovations in trial design can speed the development of new therapeutics," he explained. In recognition of his contributions, he received the Sheila Essey Award for ALS Research from the ALS Association and the American Academy of Neurology in 2014, followed by the Forbes Norris Award for ALS from the International Alliance of ALS/Motor Neuron Disease Associations in 2023.

Among his many contributions, Dr. Shefner considers the creation and growth of the Northeast ALS Clinical Trials Consortium (NEALS) to be his greatest achievement. He co-founded NEALS with Merit Cudkowicz, MD, and served as co-chair for its first 13 years. "Over 30 years, NEALS has become the largest ALS-devoted research consortium in the world, and has performed many ALS trials and biomarker studies," he said. The consortium has also contributed to education and training for physicians, research staff, patients, and caregivers, helping to unify the ALS community and accelerate progress.

Dr. Shefner's relationship with the AANEM has been a constant throughout his career. "The AANEM supported me from the start of my career and was the venue for me presenting much of my early work," he said. "To be honored by the AANEM for accomplishments in clinical care and research is one of the highlights of my career." He also emphasized his appreciation for the organization's adaptability and collegial environment.

Mentorship and collaboration have been key influences in Dr. Shefner's journey. He credits his fellowship supervisor, Christian Krarup, MD, with shaping his approach to rigorous scientific thinking and describes Merit Cudkowicz, MD, as "an invaluable collaborator."

Looking ahead, Dr. Shefner remains deeply engaged in advancing the field. He continues to lead and design clinical trials in ALS while developing new biomarkers, including fluid, physiological, and functional measures. He is particularly encouraged by recent advances in genetics. "The explosion in genetics as it relates to both diagnosis and therapy in neuromuscular disease has already led to revolutionary changes in care for multiple diseases, including ALS," he said. "I think this is only the beginning of new treatments throughout the spectrum of neuromuscular disease."

For those considering a career in the field, Dr. Shefner highlights how dramatically NM medicine has evolved. "When I first completed training, neuromuscular medicine was focused more on accurate diagnosis than curative therapies. This has completely changed, and the field is one of dramatic therapies and even cures," he said.

Despite his extensive research accomplishments, Dr. Shefner identifies patient interactions as the most rewarding aspect of his career. "As I look back, my interactions with patients have been the most rewarding part of my professional life," he shared. "The chance to meaningfully impact people's lives has positively impacted my own."

Through decades of leadership, innovation, and collaboration, Dr. Shefner has helped shape the trajectory of ALS research and NM medicine. His recognition with the AANEM Distinguished Researcher Award reflects not only his past contributions but also his continued leadership in a field moving rapidly toward new possibilities.