Morquio stunts growth in children born with ... The drug is the only treatment available for the debilitating and progressive condition, and works by replacing the N-acetylgalactosamine-6 ...

Morquio stunts growth in children born with ... which said the recommendation and continued access to the treatment "will give hope to people with MPS 4A, their families and carers of a better ...

The mucopolysaccharidosis market is witnessing a surge in innovation, driven by cutting-edge gene therapies, enzyme replacement treatments, and novel drug developments. The market is poised for ...