Aimee Donald

Aimee is a paediatric neurologist (in training) who is funded by NIHR at Manchester Foundation Trust. Her PhD focussed on the rare lysosomal storage disorder, Gaucher Disease, with a specific focus on the neuronopathic form of disease. Her work currently focuses on describing phenotype and natural history of this and other neurodegenerative conditions, primarily the neuronopathic lysosomal storage disorders to improve clinical trial design. She delivers early phase CNS-directed gene therapy trials as part of my work at MFT and hope to use my work to identify better clinical trial outcome measures. She is increasingly interested in clinical trial design, ethics and regulation, particularly in the context of accelerating access to therapies for vulnerable children with progressive disorders.