Myth Debunked: Are rare diseases too uncommon to be studied effectively?

This 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,

Researching a rare childhood form of ALS

Amyotrophic lateral sclerosis (ALS) is often thought of as a disease of adulthood, but in very rare cases, it begins in childhood or adolescence. This form, known as juvenile-onset ALS (JALS), is devastating for affected children and families. Several different genes can cause JALS when they are damaged. These genes encode proteins that are essential

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Myth Debunked: Are rare diseases too uncommon to be studied effectively?

Researching a rare childhood form of ALS

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