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BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) announced third-quarter 2025 financial results and a clinical ...

The US Food and Drug Administration (FDA) has rejected PTC Therapeutics’ new drug application (NDA) for vatiquinone in Friedreich's Ataxia. This follows the biotech’s decision to submit the ...

The FDA has issued a CRL to Biohaven regarding the NDA for troriluzole for the treatment of adults with spinocerebellar ataxia.

Voltage‐gated calcium channels, particularly the CaV2.1 channels encoded by the CACNA1A gene, are central to cerebellar function and synaptic transmission. Dysfunction in these channels leads to a ...