Ataxia with oculomotor apraxia is an autosomal recessive disease, originally described in Portuguese patients and later in Japanese patients. Symptoms first noticed appear between ages 1 and 16 ...

An FDA-approved medication can now treat Friedreich's ataxia, but health plan barriers and formulary exclusions keep many patients from receiving it.

In our rare disease themed month, we interview Alan Thomas, ataxia patient and chairman of Ataxia South Wales, on unmet needs in this space. Alan Thomas has Ataxia – a life-limiting ...

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for troriluzole for the treatment of adults with ...

Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), today announced that the US Food and Drug Administration (FDA) has ...

The annual event, held Dec. 6, 2024, at the JW Marriott Tampa Water Street, raised funds for the Friedreich’s Ataxia Research ...

Biohaven (BHVN) announced that the FDA has accepted for review the company’s New Drug Application, or NDA, for troriluzole for the treatment of ...

Quince Therapeutics (QNCX) “announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for U.S. Patent Application ...

Biohaven's NDA for Troriluzole was accepted by the FDA, potentially leading to approval in Q3 2025. Click here to find out ...

Spinocerebellar ataxia type 3 (SCA3) is a rare neuromuscular disease with a prevalence of one to five in 100,000 people. It is the most common form of spinocerebellar ataxia. The disease is caused ...

Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance ...