Rare diseases

News

Biohaven's troriluzole candidate for spinocerebellar ataxia (SCA) has been turned down by the FDA, in another sign of a tougher stance by the agency.

News

People living with thymidine kinase 2 deficiency (TK2d), a vanishingly rare and often fatal genetic disorder, finally have an approved therapy.

News

AstraZeneca's gefurulimab could give gMG patients the first treatment option that they can self-administer at home once a week.

News

BridgeBio's week just went from great to superb, as it reports a second positive topline result in a pivotal trial of a late-stage pipeline drug.

News

Germany's Merck KGaA is looking at making further acquisitions to boost its business, with a focus on life sciences and rare diseases.

News

UCB's experimental medicine for TK2d, currently under regulatory review, offers hope to patients with the devastating rare disease.