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 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 …
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.
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.
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.
Leading gene therapy company uniQure have announced preclinical data on its gene therapy candidate, AMT-150, for the treatment of Spinocerebellar Ataxia 3 (SCA3).
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.
Second trial investigating troriluzole in spinocerebellar ataxia announced by Biohaven pharmaceuticals to commence at the end of January.
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 …
