Aimee Donald, MBChB, PhD, on Making Progress With Gene Therapy in Lysosomal Storage Diseases
The pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.
Aimee Donald, MBChB, PhD, on Assessing PR001 in Children With Type 2 Gaucher Disease
The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.