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Join the Summer Match Challenge! All donations during the month of June will be matched by the Clementz-Peterson Family Fund and an anonymous donor, up to $100K. DONATE
This is a visual tool created to show the progress of SCA2 therapies that are currently being developed. Along the right side of the diagram, treatments are grouped by their mechanism, or how the drug works. The top of the diagram shows the stage of research where the drug is in development. Phases 1 through 3 involve clinical trials with human participants. Visit our PrepRARE: Clinical Trial Readiness Education page to learn more about each phase.
Once clinical trials provide enough evidence to prove that the drug is safe and effective, the sponsor of the drug can apply for regulatory approval of the drug. After regulatory approval, the drug can become available to individuals through their healthcare provider.
The agency responsible for reviewing and approving new treatments varies depending on where you are in the world. Some regulatory agencies include:
You can click the links above to learn more about each regulatory agency. This includes information about how they approve new medications.
Please note that a drug’s status is subject to change. Please visit clinicaltrials.gov for the most current information available for specific therapies. Detailed information is available for some of the drugs listed.
Troriluzole (BHV-4157, Biohaven Pharmaceuticals Inc.) is an oral prodrug formulation of Riluzole, a drug approved to treat ALS. Troriluzole helps to reduce glutamate dysfunction in the brain, which is a common symptom for many forms of ataxia. Controlling the amount of glutamate in the brain aims to improve ataxia symptoms.
Troriluzole is currently under review by the FDA to treat Spinocerebellar Ataxia (SCA).
NeuroEPO (Human-recombinant EPO, CIRAH) is an investigational drug designed by researchers at the Center for Molecular Immunology in Cuba. It is a modified version of erythropoietin, a protein that increases growth and communication between neurons.
ARO-ATXN2 (Arrowhead Pharmaceuticals/Sarepta Therapeutics) is an investigational siRNA therapy. ARO-ATXN2 prevents the creation of the ATXN2 protein, which aims to halt or slow disease progression.
Riboway Therapeutics is developing an antisense oligonucleotide (ASO) therapy SCA2. This work is currently preclinical.
Evox Therapeutics is developing a gene editing therapy SCA2. This work is currently discovery phase/preclinical.
To learn more about participating in research visit our PrepRARE Clinical Trial Readiness Education page.
