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SCAs

Reflections on the Ataxia UK Annual Conference 2023

On Friday 20th and Saturday 21st October 2023, Ataxia UK held their first ever hybrid in-person and virtual annual conference for families affected by ataxia. The conference, which took place …

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US FDA and European Commission grants VICO Therapeutics Orphan Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia 

Spinocerebellar ataxias (SCAs) are a group of rare, progressive hereditary genetic disorders that affects the cerebellum, brain stem and spinal cord. Currently, more than 30 types of SCAs have been identified. SCAs are caused by genetic mutations, …

US FDA and European Commission grants VICO Therapeutics Orphan Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia  Read More »

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 …

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

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

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

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