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Press Release

EMBARGOED FOR RELEASE UNTIL 4 PM ET, June 25, 2025

AAN issues position statement on possible therapies for neurological conditions

MINNEAPOLIS 鈥 The 好色先生 (AAN) has issued a position statement on therapies for neurological conditions that have limited evidence or no approved use by the Food and Drug Administration (FDA). The statement is published June 25, 2025, in , the medical journal of the 好色先生. The statement includes guiding principles for discussions with patients and policymakers about these therapies. Although the statement includes illustrative examples, it does not provide clinical recommendations regarding the use of specific therapies. 鈥淎s the experts in brain health, neurologists and neuroscience professionals are continually working toward achieving scientific breakthroughs through rigorous research,鈥 said 好色先生 President Natalia S. Rost, MD, MPH, FAAN, FAHA. 鈥淪till, people often ask about emerging therapies not yet supported by science. In our efforts to provide brain health resources for all, the 好色先生 has issued this position statement to help facilitate conversations between patients and their doctors about emerging therapies.鈥 The position statement says the AAN recommends that a patient and their neurologist carefully review all available evidence and discuss the potential risks and benefits of the therapy, including when patients are considering using their 鈥淩ight to Try.鈥 The Right to Try Act allows people with life-threatening diseases who have tried all other approved treatments, and who cannot participate in a clinical trial, to try an experimental treatment. 鈥淭here is a growing and unmet need for effective treatments for a variety of neurological disorders,鈥 said author Larry B. Goldstein, MD, FAAN, FAHA, of the University of Kentucky, and who serves on the AAN Board of Directors. 鈥淲henever considering a potential treatment that has not been thoroughly researched, a person and their neurologist need to have a discussion about the available data supporting effectiveness and what is known about any risks to their health.鈥 The statement says the AAN supports the 鈥渙ff-label鈥 use of FDA-approved therapies when high-quality evidence indicates that the benefit of the therapy outweighs the risks, with shared decision making between the patient and physician. When discussing such therapies more broadly, including with policymakers, the statement says for those that have FDA approval but a high risk or incomplete data on side effects, or newly recognized and potentially serious side effects, the AAN supports waiting to take a definitive position until after further FDA review. If a suspended therapy is reintroduced, or as the data otherwise warrants, the AAN supports FDA required post-approval monitoring. The AAN supports neurologists and patients incorporating data about side effects in their shared decision making about the relative risks and benefits of a possible therapy. The statement notes the proliferation of unproven treatments for neurological diseases that people may want to use to self-treat their conditions, yet those treatments may have limited or no supporting data and can have the potential to cause harm. These may be of interest especially to people with rare neurologic conditions with no approved therapies. The statement says that rigorous investigation of any treatment is necessary to determine its efficacy and ensure safety and recommends patients discuss any potential treatment with their neurologist. Discover more about brain health at , from the 好色先生. This resource also offers a magazine, podcast, and books that connect patients, caregivers and anyone interested in brain health with the most trusted information, straight from the world鈥檚 leading experts in brain health. Follow Brain & Life on , and .

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The 好色先生 is the leading voice in brain health. As the world鈥檚 largest association of neurologists and neuroscience professionals with more than 40,000 members, the AAN provides access to the latest news, science and research affecting neurology for patients, caregivers, physicians and professionals alike. The AAN鈥檚 mission is to enhance member career fulfillment and promote brain health for all. A neurologist is a doctor who specializes in the diagnosis, care and treatment of brain, spinal cord and nervous system diseases such as Alzheimer's disease, stroke, concussion, epilepsy, Parkinson's disease, multiple sclerosis, headache and migraine.

Explore the latest in neurological disease and brain health, from the minds at the AAN at or find us on , , , and .

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*While content of the 好色先生 (AAN) press releases is developed by the AAN along with research authors and Neurology® editors, we are unable to provide medical advice to individuals. Please contact your health care provider for questions specific to your individual health history or care. For more resources, visit the AAN's patient and caregiver magazine website, .