CHARLOTTE, N.C. — Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a devastating disease that takes away a person's ability to move and speak. Now, doctors say a brand-new approach to treatment is showing surprising results
In a recent breakthrough, a drug was custom-built for a patient with a rare genetic mutation. It works by shutting down the faulty gene that causes damage to nerve cells.
In one case, the disease slowed down so much that a woman regained the ability to walk and breathe on her own. In another case, a man who started treatment before symptoms even showed up is still healthy — three years later.
This is what doctors call " N-of-1 "" medicine: therapies designed for just one person. Now, the success of these early cases has led to a global t