Measuring What Matters in ALS: The ALSFRS-R

The ALS Functional Rating Scale–Revised (ALSFRS-R) has become one of the most essential tools for measuring functional decline in amyotrophic lateral sclerosis (ALS). Used for more than two decades across clinical research, drug development, and patient care, the ALSFRSR provides a standardized method for evaluating patients’ abilities in everyday activities and predicting key  clinical outcomes, including survival. While the ALSFRS-R is a wellestablished and broadly accepted tool, it remains dynamic, evolving in line with advances in the ALS community’s  understanding of disease heterogeneity, functional milestones, and contemporary patient priorities.

This webinar will examine the history, rationale, development, and validation of the ALSFRS-R, including its origins as a simpler, questionnaire-based alternative to earlier functional scales. Attendees will gain insight into how the ALSFRS-R was constructed, the principles that guided its design, and the robust evidence supporting its internal consistency, cross-sectional and  longitudinal validity, and strong correlation with measures such as muscle strength, respiratory function, and self-reported dependency.

At the same time, the session will address critical limitations and challenges that researchers and clinicians must consider. These include inter-patient variability, floor and ceiling effects, non-linearity in late disease stages, outdated item wording, and concerns around domain structure and unidimensionality. Particular emphasis will be placed on the ALSFRS-R’s respiratory domain, which demonstrates weaker correlation with other domains and underscores opportunities for scale refinement.

Looking ahead, the webinar will explore emerging efforts to standardize the ALSFRS-R administration globally, improve linguistic and cultural consistency, and incorporate modern technologies such as remote assessments and digital health technologies. Opportunities for expanding the scale to better capture cognition, pain, mood, and other patient-reported experiences will also be discussed.

By the end of the session, attendees will have a comprehensive understanding of the ALSFRS-R’s strengths, weaknesses, and future directions, equipping them to use the scale more effectively in clinical trials, real-world practice, and multidisciplinary care.

Speakers

Jesse M. Cedarbaum, MD, FAAN (Neurology), FANA, Professor of Neurology, Yale School of Medicine

Dr. Jesse M. Cedarbaum is a Neurologist whose career has focused on developing novel and improved therapeutic agents for neurodegenerative and neuropsychiatric disorders. He has a unique background of more than 30 years of drug development and clinical trials experience across academia, biotechnology and large and small pharmaceutical companies. Most recently, he led a team at Biogen that advanced three new compounds for potential treatment of Parkinson’s disease into the clinic, efforts that included development of novel biomarkers and digital health technologies. He is also the creator of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) and the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), which stratifies the severity of ALS, including respiratory function.

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Katie Flynn, Account Executive, Mapi Research Trust

Katie Flynn brings over a decade of experience in Account Management and International Business Development, with a strong background in Client Services. As an Account Executive at the Mapi Research Trust, she focuses on building new partnerships with authors and copyright holders in Clinical Outcome Assessments and supporting them in the day-to-day management of their questionnaires.

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