Myth Debunked: Are rare diseases too uncommon to be studied effectively?
2This is a common misconception ‒ and a costly one. Although juvenile-onset ALS (JALS) affects few individuals, its rarity is what makes it scientifically powerful. Many JALS cases are caused by single-gene mutations, providing a clear genetic entry point to understand how and why motor neurons degenerate. Unlike more common diseases with complex causes,