Research News - Ataxia UK

Research News

In conversation with the London Ataxia Centre Speech and Language Therapist, Lucie Hogger

Recently, our research intern Scarlett Parr-Reid sat down with Lucie Hogger, Speech and Language Therapist at the London Ataxia Centre to talk about the speech and language therapy services offered […]

In conversation with the London Ataxia Centre Speech and Language Therapist, Lucie Hogger Read More »

Intrabio announces FDA approval of their drug Levacetylleucine (Aqneursa) for the treatment of Niemann-Pick Disease Type C

On 25th September 2024, the pharmaceutical company Intrabio announced that the FDA in the US has approved their small molecule drug Levacetylleucine for the treatment of Niemann-Pick Disease Type C

Intrabio announces FDA approval of their drug Levacetylleucine (Aqneursa) for the treatment of Niemann-Pick Disease Type C Read More »

Vico Therapeutics announces positive interim data in their phase 1/2a trial of VO659, which is being tested in people with Huntington’s disease (HD) and spinocerebellar ataxias 1 and 3

On September 13th, the pharmaceutical company Vico Therapeutics announced positive interim phase 1/2a clinical data of VO659 in the treatment of Huntington’s disease. VO659 demonstrated a 28% average reduction in

Vico Therapeutics announces positive interim data in their phase 1/2a trial of VO659, which is being tested in people with Huntington’s disease (HD) and spinocerebellar ataxias 1 and 3 Read More »

Satisfaction is significantly higher in those attending UK specialist ataxia centres compared with those who do not, new paper shows

Ataxia UK has worked closely with the London Ataxia Centre researchers and other experts on a research study assessing the impact of specialist ataxia centres in Europe. This was part

Satisfaction is significantly higher in those attending UK specialist ataxia centres compared with those who do not, new paper shows Read More »

Lexeo announces positive results from their phase 1/2 clinical trial programme for FA cardiomyopathy

Lexeo Therapeutics is a pharmaceutical company developing a gene therapy called LX2006 for people with Friedreich’s ataxia (FA) hypertrophic cardiomyopathy (disease of the heart involving stretching, thickening, or stiffening of

Lexeo announces positive results from their phase 1/2 clinical trial programme for FA cardiomyopathy Read More »

Ataxia UK hosts an educational webinar on the UK medicines approval process

On 12th July Ataxia UK hosted a webinar on how medicines get approved for use in the UK and how decisions are made on their funding in the NHS. The

Ataxia UK hosts an educational webinar on the UK medicines approval process Read More »

In conversation with Dr. Federico Herrera, Group Leader of the Cell Structure and Dynamics (CSD) Lab from the Biosystems and Integrative Sciences Institute (BioISI) at the Faculty of Sciences of the University of Lisbon (Ciências ULisboa).

Our research intern, Scarlett Parr-Reid, sits down with Dr. Herrera to discuss his research on spastic ataxia type-8 (SPAX8). Can you share a little about yourself, your background as a

In conversation with Dr. Federico Herrera, Group Leader of the Cell Structure and Dynamics (CSD) Lab from the Biosystems and Integrative Sciences Institute (BioISI) at the Faculty of Sciences of the University of Lisbon (Ciências ULisboa). Read More »

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Larimar Therapeutics announces progress of the Nomlabofusp (CTI-1601) trial for Friedreich’s ataxia

Following clearance by the US drug regulatory body the Food and Drug Administration (FDA) in July 2023, the pharmaceutical company Larimar Therapeutics has completed full enrolment and dosing of the

Larimar Therapeutics announces progress of the Nomlabofusp (CTI-1601) trial for Friedreich’s ataxia Read More »

Alterity Therapeutics presents data on the potential of ATH434 to treat FA

The biotechnology company Alterity Therapeutics presented data from their lead Friedreich’s ataxia (FA) drug candidate ATH434 at the 2024 US World Orphan Drug Congress.  In FA, a genetic change in

Alterity Therapeutics presents data on the potential of ATH434 to treat FA Read More »

An interview with Netherlands-based PhD researcher Ilse Willemse, on the SPAX app for home monitoring in ARSACS and SPG7 as part of the European PROSPAX project 

The PROSPAX (PROgression chart of SPAstic ataXias) project focuses on ARSACS and SPG7, and is a collaboration between neurologists in several sites across Europe and Canada. The overall aim is to gain

An interview with Netherlands-based PhD researcher Ilse Willemse, on the SPAX app for home monitoring in ARSACS and SPG7 as part of the European PROSPAX project  Read More »

Voyager Therapeutics and Neurocrine Biosciences announce selection of a lead gene therapy candidate for their FA program

On 26th February, the biotechnology company Voyager Therapeutics announced that as part of their collaboration with the biopharmaceutical company Neurocrine Biosciences, they have selected a lead development candidate in their

Voyager Therapeutics and Neurocrine Biosciences announce selection of a lead gene therapy candidate for their FA program Read More »

The PROSPAX Consortium publishes an infographic on their natural history study of individuals with ARSACS and SPG-7 in Europe

The PROSPAX (PROgression chart of SPAstic ataXias) project is a collaborative effort between neurologists across several sites throughout Europe and Canada. The aim is to better understand how spastic ataxias

The PROSPAX Consortium publishes an infographic on their natural history study of individuals with ARSACS and SPG-7 in Europe Read More »

New study shows impacts of FA on daily life, wellbeing, and relationships in those with FA and their caregivers  

Findings from a recent study, ‘Health-related quality of life in Friedreich Ataxia: A study of impacts on patients and caregivers’, were presented at the International Society for Pharmacoeconomics and Outcomes

New study shows impacts of FA on daily life, wellbeing, and relationships in those with FA and their caregivers   Read More »

Paper published on study of effectiveness of omaveloxolone for Friedreich’s ataxia using natural history study data

The natural history of Friedreich’s ataxia (FA) has been studied as part of a project called the Friedreich’s ataxia Clinical Outcome Measures Study (FACOMS). Natural history studies track the course

Paper published on study of effectiveness of omaveloxolone for Friedreich’s ataxia using natural history study data Read More »

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