Research News - Ataxia UK

Research News

The Sheffield Ataxia Centre’s 2025 ataxia study day for healthcare professionals

Last month, on 7th November, the Ataxia UK-accredited Sheffield Ataxia Centre hosted an ataxia study day for healthcare professionals at the University of Sheffield, chaired by Consultant Neurologists Professor Marios Hadjivassiliou and Dr Priya Shanmugarajah, and organised

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Sharing results from Ataxia UK-funded project on restoring NKX6-2 gene function in Spastic Ataxia 8 cells in the lab

Dr Federico Herrera of the University of Lisbon, Portugal, has recently shared results of his Ataxia UK-funded project, ‘Restoring NKX6-2 function by protein complementation: a proof-of-concept’. Spastic ataxia 8 (SPAX8)

Sharing results from Ataxia UK-funded project on restoring NKX6-2 gene function in Spastic Ataxia 8 cells in the lab Read More »

New paper published on TG6 antibodies and patient outcomes in Gluten-related neurological disorders

Gluten-related disorders, also known as gluten sensitivity disorders, happen when a person’s immune system reacts to gluten, and immune cells called autoantibodies, mistakenly attack the body’s tissues. This is called an autoimmune response. There are numerous autoantibodies that can be

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Solid Biosciences granted FDA rare paediatric disease designation for their FA gene therapy

The pharmaceutical company Solid Biosciences announced in December that their gene therapy for Friedreich’s ataxia (FA),  known as SGT-212, has been granted Rare Pediatric Disease and Fast Track designations by

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Ataxia UK-funded project shows that mitochondrial proteins may be a potential target to treat cellular stress in Friedreich’s ataxia

Dr Rosella Abeti and Prof Paola Giunti at the London Ataxia Centre, UCL Institute of Neurology, have shared results from their project which was funded by Ataxia UK. The project was titled ‘Exploring novel iron-mediated mechanisms to prevent cellular death in

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Larimar Therapeutics Announces positive results from their phase II trial of Nomlabofusp in FA

On 29th September 2025, the pharmaceutical company Larimar Therapeutics announced positive results from their phase II trial of the drug Nomlabofusp for FA.  Nomlabofusp is designed to increase levels of

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Spastic Ataxia Composite (SPAXCOM): a scale to evaluate disease progression in people with spasticity and ataxia

Current clinical rating scales to measure disease progression in people with spasticity and ataxia are focused solely on either spasticity symptoms (stiffness of muscles leading to involuntary spasms) or ataxia

Spastic Ataxia Composite (SPAXCOM): a scale to evaluate disease progression in people with spasticity and ataxia Read More »

PTC Therapeutics announces that the FDA has not approved Vatiquinone to treat FA in adults and children

On 19th August 2025, the pharmaceutical company PTC Therapeutics announced the disappointing news that the US drug regulators the FDA have not approved the drug Vatiquinone to treat adults and

PTC Therapeutics announces that the FDA has not approved Vatiquinone to treat FA in adults and children Read More »

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