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 […]
On September 23rd, 2024, the pharmaceutical company Biohaven announced positive topline results from their study of the drug Troriluzole to treat SCAs 1,2,3,6,7,8 and 10. Those with SCA who
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
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 »
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 »
On October 26th 2023, the pharmaceutical company Biohaven submitted an application for a new drug approval of Troriluzole to the European Medicines Agency (EMA) for the treatment of Spinocerebellar Ataxia
Biohaven submits application to the EMA for a new drug approval of Troriluzole for SCA3 Read More »
On 1st December 2023, the pharmaceutical company Seelos Therapeutics announced they had stopped their phase 2/3 programme known as STRIDES, which had been investigating a new drug for Spinocerebellar Ataxia
Seelos Therapeutics has stopped their phase 2/3 trial of Trehalose for SCA3 Read More »
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
Reflections on the Ataxia UK Annual Conference 2023 Read More »
Biohaven is a pharmaceutical company developing Troriluzole as a treatment for SCA3. In May 2023, Biohaven submitted a New Drug Application (NDA) to the US regulators (the FDA), to apply
Biohaven update on Troriluzole programme Read More »
Biohaven is a pharmaceutical company with an ataxia research programme. They carried out a clinical trial testing Troriluzole in a number of SCAs. Results of this 48 week trial showed
Biohaven submits NDA to FDA Read More »
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,
Biogen announce they have stopped SCA3 clinical trial Read More »
Vico Therapeutics is a company developing therapies for neurological conditions, including some of the SCAs. In April 2023, they announced that they have dosed the first patient in their Phase
Vico Therapeutics – First patient dosed in trial Read More »
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,
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
Leading gene therapy uniQure have published data on their gene therapy candidate AMT-150.
uniQure publishes data from spinocerebellar ataxia type 3 gene therapy study Read More »
Earlier this year the FDA granted an Orphan Drug Designation to an investigational treatment developed by Cadent Therapeutics for spinocerebellar ataxia. They plan to begin a clinical trial by the end of 2019.
Cadent Therapeutics aims to begin new trial in SCAs by the end of 2019 Read More »
Spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) are caused by mutations in the Ataxin-3 and Ataxin-1 genes that. Researchers recently tested out a type of therapy that works by targeting these genes.
Toxic protein production reduced in SCA3 and SCA1 by genetic approach Read More »
Leading gene therapy company uniQure have announced preclinical data on its gene therapy candidate, AMT-150, for the treatment of Spinocerebellar Ataxia 3 (SCA3).
uniQure releases new data from Spinocerebellar Ataxia Type 3 gene therapy study Read More »
The ‘New Modalities Ecosystem’, launched in 2018 by the Finnish pharmaceutical company Orion, aims to exploit Finnish expertise to deliver new treatments and technologies for rare diseases.
New initiative for rare diseases launched by Finnish pharmaceutical company Read More »
Second trial investigating troriluzole in spinocerebellar ataxia announced by Biohaven pharmaceuticals to commence at the end of January.
SCA drug trial to begin in America Read More »
Catch up on the latest research news!
Research News – May and June 2018 Read More »
IntraBio, a U.K. based biopharmaceutical company, has received an Orphan Drug Designation from the US Food and Drug Administration (FDA) for its lead compound (IB1000) for the treatment of spinocerebellar
FDA Orphan Drug Designation for Spinocerebellar Read More »
Catch up on the Autumn’s latest research news.
Research News – October and November 2017 Read More »
Read up on the latest research news!
Latest Research News – January and February 2017 Read More »
