MLD is a rare, genetic, degenerative, neurometabolic disorder that affects 1 in 40,000 people worldwide.

There are up to 70 different mutations in the GAA gene that cause the symptoms of Pompe disease.

A new wave of treatments based on gene editing technology are expected to be made available over the next few years. 

Distal muscular dystrophy is a general term for a group of rare progressive genetic disorders.

What are the changes pharmaceutical companies need to be aware of and how to manage them?

The objective of this paper is to address clinical trials inclusion in the Middle East.