Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...

Good morning! Today, we get deeper into the ousting of Nicole Verdun at the FDA, see promise from Vertex’s cell therapy for ...

Capricor stock drops as FDA cancels advisory meeting on Duchenne therapy; leadership changes and regulatory tensions fuel uncertainty.

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and ...

Bernstein has high conviction in positive outcomes but views Avidity’s DM1 phase 3 readout as the primary stock catalyst, citing ongoing regulatory uncertainty for Dyne. In DM1, Bernstein models ...

Analysts have recently evaluated WAVE Life Sciences and provided 12-month price targets. The average target is $16.0, ...

On Friday 27 June, an inspiring group of cyclists will set off from Greenwich, London, on an ambitious 300-mile journey to ...

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.

This story was excerpted from Bryan Hoch’s Yankees Beat newsletter. To read the full newsletter, click here. And subscribe to ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 ...

The FDA had resolved the shortage of Wegovy in April, and telehealth providers were advised to stop selling compounded ...