FDA takes the bull by the horns on rare neurodegenerative diseases

The US Food and Drug Administration (FDA) has unveiled an Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS).

This five-year strategy seeks to improve and extend the lives of people living with rare neurodegenerative diseases by advancing the development of new drugs and improving access to them.

FDA Commissioner Robert Califf said: “The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients. We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases.