Award for researcher behind first treatment for rare disease

A UCLH and UCL researcher who helped deliver the first-ever treatment for the rare neurodegenerative disease Friedrich’s ataxia has won a UCL award for her collaboration with industry to bring the treatment to patients.

Professor Paola Giunti, who has been supported by the NIHR UCLH Biomedical Research Centre, received the ‘Impactful Partnership with Industry’ award at a ceremony earlier this month, for the instrumental role her team had in validating the drug, and the global clinical trial of the drug alongside Reata Pharmaceuticals – now Biogen.

The drug, known as Omav, gained approval for use in the United States in 2022, becoming the first approved treatment for Friedrich’s ataxia (FA), with European approval following in 2024. UK regulators are currently assessing the drug.

FA is a rare, inherited neurodegenerative disease, affecting about 22,000 people worldwide. It typically emerges in childhood and causes progressive loss of coordination, muscle weakness, and fatigue, often leading to wheelchair reliance, and premature death.

The trial recruited participants from the US, Europe, and Australia. Patients at UCLH took part at the NIHR Clinical Research Facility at UCLH.

Prof Giunti, who is Head of the UCLH Ataxia Centre, said:

"I am very honoured to be recognised by this award and to have experienced such a longstanding partnership with industry to impact the life of my patients. I always work with patients who have been at the centre of my studies and clinical service, and to see them directly benefit from this research that came directly from the Ataxia Centre is what gives me my drive and purpose."

Find out more about the award: www.ucl.ac.uk