Riluzole is the only agent approved as a disease-modifying treatment for motor neurone disease (MND). It may, or may not (depending on how one views the evidence), modify the rate of disease progression. We will examine the use of this drug that is generally viewed to have at best only a limited impact on survival in a miserable disease. With the general neurologist in mind, we hope to present a balanced view of the available evidence, with thoughts for future research and what we do in our own practice. We will highlight some of the scientific and ethical issues posed by disorders like MND, and the constraints imposed by the priorities of the pharmaceutical industry and state-sponsored research bodies.
WHAT IS THE IDEAL?
What then are the desirable features of a disease-modifying therapy for MND? We propose the following:
significant increase in survival – at least 4 months (i.e.
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