Dr. P. Fratta
The group focuses on motor neuron diseases (MND), specifically on amyotrophic lateral sclerosis (ALS) and spinal bulbar muscular atrophy (SBMA).
We use mouse models and in vitro systems to understand basic pathogenic mechanisms and are investigating alterations in RNA metabolism, with a focus on axonal RNA.
We are also carrying out clinical research with the goal of developing biomarkers and better outcome measures for disease progression, which are vital for future clinical trials.
We are currently funded by:
UK Medical Research Council
Motor Neurone Disease Association
UCLH NIHR Biomedical Research Centre
Kennedy’s Disease UK
The Rosetrees Foundation