Research News – Ataxia

Research News

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Larimar Therapeutics: FA Drug Update

Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, such as Friedreich’s ataxia (FA). Previously, we reported that Larimar Therapeutics were evaluating the FA drug CTI-1601 in a Phase 1 multiple ascending dose clinical trial (https://www.ataxia.org.uk/research-news/larimar-therapeutics/). FA is caused by a fault in the frataxin gene, which means that lower levels of frataxin are produced. CTI-1601 is …

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New paper published on lower urinary tract and bowel dysfunction in spinocerebellar ataxias

A new paper from the team at the London Ataxia Centre provides new information on symptoms of the lower urinary tract (LUT) and the bowel in people with spinocerebellar ataxias (SCAs). The lower urinary tract consists of the bladder and the urethra (the tube carrying urine from the bladder to outside the body).  The objective of this research was to evaluate the prevalence of LUT …

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New Ataxia Consortium created to accelerate the development of treatments for the Ataxias

Ataxia UK, along with Ataxia Global Initiative and National Ataxia Foundation, recently partnered with Critical Path Institute (C-Path) for the launch of their Critical Path to Therapeutics for the Ataxias (CPTA) Consortium. CPTA is a public-private partnership focused on accelerating the development of treatments for inherited Ataxias. The work of the Consortium will focus on …

New Ataxia Consortium created to accelerate the development of treatments for the Ataxias Read More »

DRPLA Patient Registry

Ataxia UK and the US Foundation CureDRPLA are the lead organisations of the CureDRPLA Global Patient Registry. This Registry aims to collect data on as many Dentatorubral-pallidoluysian atrophy (DRPLA) patients as possible. The Registry collects patient-reported data on individuals affected with DRPLA once a year to understand how the condition changes over time. Having a …

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Neuromodulation of the cerebellum rescues movement in a mouse model of ataxia

The International Neuromodulation Society defines therapeutic neuromodulation as “the alteration of nerve activity through targeted delivery of a stimulus, such as electrical stimulation or chemical agents, to specific neurological sites in the body.” An example of neuromodulation therapy is deep brain stimulation (DBS). DBS is a neurosurgical technique, that requires the implantation of electrodes into specific areas in the …

Neuromodulation of the cerebellum rescues movement in a mouse model of ataxia Read More »

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 United States’ Food and Drug Administration (FDA) to hear directly from patients, their families, caregivers, and patient advocacy organisations. This meeting focused on polyglutamine spinocerebellar ataxias …

The Voice of the Patient: Living with Polyglutamine Spinocerebellar Ataxias and Dentatorubral-Pallidoluysian Atrophy Read More »

Update from Novartis Gene Therapies on treatment for FA 

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 announced that the US Food and Drug Administration (FDA), which is the drug regulatory body in the US, has granted Orphan Drug Designation for OAV401. Orphan Drug Designation is …

Update from Novartis Gene Therapies on treatment for FA  Read More »

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 Disease Strategy, has been designed around the views and experiences of those living with rare diseases. The rare-disease community helped to identify 4 key priority areas to bring …

UK Rare Disease Framework launched to improve diagnosis and treatment of rare diseases Read More »

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