THE IMPORTANCE OF NATURAL HISTORY data IN SOLVING RARE DISEASES

"For any disease in a rare disease population, for which you're trying to show that your drug or gene therapy actually works, you have to understand the disease first.  And so, you need what's called Natural History data.  That is crucial in the rare disease space" - Dr. Rebecca Ahrens MD, metabolic physician at Children's Hospital of Philadelphia (CHOP).

In this 3-minute clip (below), Dr. Ahrens explains the vital importance of having natural history data and why gathering that data is particularly challenging for an ultra-rare, neurodegenerative disease like Sanfilippo Syndrome.

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"You have to understand the disease first. And so, you need what's called Natural History data. That is crucial in the rare disease space"

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To watch the full conversation (22 minutes long) with Dr. Ahrens, click here: