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Updated: Sep 18, 2025

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

Principles for Novel Neurologic Therapeutics: An AAN Position Statement.

Larry B Goldstein¹, Jonathan R Crowe^2,3, Anindita Deb⁴

¹Department of Neurology, University of Kentucky, Lexington.

|June 25, 2025

Summary

This summary is machine-generated.

The American Academy of Neurology (AAN) offers guidance for policymakers on novel therapies with limited evidence. Neurologists and patients should discuss risks and benefits, especially when considering "right to try" options.

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Area of Science:

Neurology
Health Policy
Evidence-Based Medicine

Background:

The American Academy of Neurology (AAN) provides guidance on therapies with potential neurologic benefits but limited evidence or no FDA approval.
This includes situations where patients consider accessing treatments under

Purpose of the Study:

To outline the AAN's principles for advising policymakers on therapies with limited scientific validation.
To guide neurologists and patients in evaluating novel treatments with uncertain efficacy and safety profiles.

Main Methods:

This is a policy statement outlining general principles.
It involves expert consensus and review of existing evidence frameworks.

Main Results:

The AAN recommends a collaborative approach between neurologists and patients.
This involves a thorough review of available data and a discussion of potential risks and benefits.

Conclusions:

Careful consideration and informed discussion are crucial when evaluating therapies with limited evidence.
The AAN emphasizes patient-centered decision-making in complex therapeutic scenarios, including "right to try" considerations.