At Denali Therapeutics, where I serve as chief medical officer and head of development, we are eager to develop new treatments for progressive and debilitating neuronopathic mucopolysaccharidoses diseases with our novel technology. We are not alone—several other biopharma companies also have drug candidates that have shown promise in clinical testing. Unfortunately, as so many families and physicians know, the road to approval of new treatments for rare diseases is long and slow.

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