rare diseases

Crossing crosswalk walking road

Biotech

Ionis rare disease candidate ekes out phase 3 win

Out of 54 patients with Alexander disease, those given zilganersen could walk an average of 33.3% faster in a 10-meter walk test at 61 weeks.

Darren Incorvaia Sep 22, 2025 10:46am

road through forest

Fierce Pharma

Stealth scores FDA approval for 1st Barth syndrome treatment

Sep 19, 2025 6:00pm

Businessman climbing steps to pluck a star from the sky

Regeneron's ultrarare disease phase 3 hits, teeing up filings

Sep 17, 2025 9:31am

Network of blood vessels against a white background

Custom gene editor fixes mutation causing deadly disease in mice

Sep 11, 2025 1:48pm

patent infringement patent lawsuit

Arrowhead sues Ionis over patent dispute for Tryngolza rival

Sep 11, 2025 11:33am

pause button on blue background

Capsida pauses phase 1 gene therapy trial after child dies

Sep 11, 2025 7:30am