Research Impact Report 2023
The research that Ataxia UK funds has a real impact on the world of ataxia research, and our understanding of all ataxias. Every year we ask Ataxia UK-funded researchers to […]
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Results from PTC Therapeutics Friedreich’s ataxia trial
PTC Therapeutics have announced the initial results of their Phase 3 MOVE-FA trial of vatiquinone (PTC-743) in people with FA. Vatiquinone targets an enzyme called 15-lipoxygenase, which is a regulator
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Newly discovered ataxia SCA27B shows promising response to a treatment
Late-onset spinocerebellar ataxia 27B (SCA27B) is newly discovered ataxia which is caused by a mutation in a gene called fibroblast growth factor 14 (FGF14). This discovery may lead to more
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Preliminary results of Larimar Therapeutics FA trial show increase in frataxin levels
Larimar Therapeutics is a biotechnology company, developing a treatment for Friedreich’s ataxia (FA) called CTI-1601. CTI-1601 replaces frataxin, which is the protein that is reduced in FA. More specifically, CTI-1601
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Biogen announce they have stopped SCA3 clinical trial
Ataxia UK were saddened to hear that Biogen have stopped their Phase I clinical trial for SCA3 therapeutic BIIB132 (MERA study). The trial was planned to be an international study,
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New genetic cause of late-onset ataxia discovered
Many people with ataxia do not have a specific genetic diagnosis and the cause of their ataxia is unknown. Two papers have been published which have identified a genetic mutation
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Covid-19 Booster updateÂ
There are currently 3 types of boosters for the Covid-19 vaccine:Â A 1st booster dose of the COVID-19 vaccine: Everyone aged 16 and over who had their 2nd dose vaccine
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Paper published on the attitudes of people with ataxia towards clinical trials
In January 2022, researchers at UCL/UCLH, Children’s Hospital of Philadelphia, Ataxia UK and Friedreich’s Ataxia Research Alliance (FARA), published a paper on the attitudes of people with ataxia towards clinical
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Update on Novartis Gene Therapies Friedreich’s ataxia Program
Please see below an update from Novartis Gene Therapies on their Friedreich’s ataxia program: February 2022 Novartis Gene Therapies had been developing a therapeutic approach for Friedreich’s ataxia. One candidate,
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Eligibility for Covid-19 treatments
Updated: June 6, 2022 Coronavirus Treatments The COVID-19 vaccines continue to remain the best way to defend against a coronavirus infection. However, for some people with certain health conditions there
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Ataxia UK-funded project develops new TTBK2 cell models
In their Ataxia UK-funded research project, Dr Mariana Santos Moreda Graça and her team at the Institute for Molecular and Cell Biology (IBMC, Portugal) developed new cell models to help
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Larimar Therapeutics: FA Drug Update
Update 27/09/2022 In May 2021, the US Food and Drug Administration (FDA) put a full clinical hold on Larimar Therapeutics programme for Friedreich’s ataxia (FA) drug CTI-1601. CTI-1601 is a
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Covid-19 vaccine and gene therapy
Vaccines currently approved in the UK At present, there are three Covid-19 vaccines approved for use in the UK: Pfizer/BioNTech Oxford/AstraZeneca Moderna How do these vaccines work? Messenger RNA (mRNA) vaccines: Both the Pfizer/BioNTech and Moderna
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The Voice of the Patient: Living with Polyglutamine Spinocerebellar Ataxias and Dentatorubral-Pallidoluysian Atrophy
On September 25, 2020 the National Ataxia Foundation (NAF) and CureDRPLA organised the Externally-Led Patient Focus Drug Development (EL-PFDD) meeting. You can watch the recording here. The EL-PFDD meeting provided a unique opportunity for the
Update from Novartis Gene Therapies on treatment for FAÂ
Novartis Gene Therapies have announced an update on a gene therapy that they have been developing for Friedreich’s Ataxia (FA) called OAV401.  Novartis Gene Therapies develops and commercialises gene therapies for rare genetic conditions, such as FA. They have
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Covid-19 vaccine priority groups
Ataxia UK has recently been made aware that some people with ataxia are not being ‘flagged up’ on their GP’s systems as being in the appropriate priority group for the Covid-19 vaccine. Our Medical Advisory Panel strongly advise
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UK Rare Disease Framework launched to improve diagnosis and treatment of rare diseases
A new government strategy to improve the lives of people living with rare diseases, across the 4 nations of the United Kingdom, has now been launched. The UK Rare Disease Framework, following on from the previous Rare
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FA drug clinical trial update: Larimar TherapeuticsÂ
Recently, Ataxia UK had a meeting with representatives from Larimar Therapeutics, a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, to discuss possible collaborations to help progress
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