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ALS and many of the other neurodegenerative disorders have not received considerable attention from regulated industry. This fact is likely due in part to the logistical difficulties involved in studying them. However, another one is the perceived lack of market opportunity in this area. The relatively small size of the populations for disorders such as ALS and Huntington’s disease, or rare disorders such as Cockayne syndrome or ataxia-telangiectasia have been offputting to companies that normally measure their target patient populations in the high hundreds of thousands or millions. Medical products such as the cancers or those responding to antibiotics fit into this category.

An important attribute of most cancer chemotherapy or antibiotic use is that these drugs that are used for a relatively short period of time by a patient, and, ideally, never again. Thus, although the target patient population is very large, the short periods of use for each drug limit their use. Alternatively, any truly effective treatment for a neurological disorder will require lifelong use after diagnosis. Thus, the length of time of use for an ALS treatment would more than compensate for the size of the patient population. The success of Genzyme’s Cerezyme more than proves this point. Cerezyme has become the gold standard for the treatment of Type I Gaucher’s disease, and is used by 4,800 patients around the world. In spite of the very small size of the patient population, Cerezyme is Genzyme’s top selling product.