Biohaven's troriluzole candidate for spinocerebellar ataxia (SCA) has been turned down by the FDA, causing shares in the company to lose more than 40% of their value in pre-market trading. The news is ...

LEXINGTON, Mass., March 09, 2026 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today reported ...

Low dose alnodesertib plus gemcitabine met the primary endpoint of progression-free survival (PFS) versus gemcitabine alone in patients with platinum-resistant high-grade serous ovarian cancerStudy ...

Rare Disease Day, annually held on the last day of February, is a day intended to increase awareness on rare diseases that impact people’s lives globally, and to celebrate the meaningful contributions ...

Kolkata: For the first time in India, a team of city neurologists and researchers created ‘mini brains' out of real human tissues and blood vessels to.

Severe neurodegeneration is one of several clinical features of ataxia-telangiectasia (AT), yet control of CNS symptoms in patients with AT has received little attention. Buoni et al. have presented a ...

A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the agency’s bureaucracy affects innovation, with one witness testifying that ...

After an FDA rejection of its spinocerebellar ataxia (SCA) therapy, Biohaven was forced to rethink its strategy—cutting R&D spend by 60% and channeling the spirit of a scrappy startup again as it ...

National Poison Prevention Month is coming up in March, so we thought it was timely and important to highlight this case for ...

Rare genetic diseases such as ataxia telangiectasia and cystic fibrosis lack strong commercial incentives, prompting charities to fund early drug research. Innovative approaches are offering renewed ...

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