Rare diseases

Patients

A look at the benefits of genetic testing in diagnosis of rare kidney diseases, from HealthLumen.

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.