The U.S. FDA has paused Rocket Pharmaceuticals' mid-stage gene therapy trial after a patient died from serious complications, ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

The death of a patient in a trial of an experimental gene therapy casts new shadows over a technology once seen as the future ...

The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, ...

A 5-year-old boy from Center City has faced rare odds since birth and now this warrior is in a real life race to rewrite his story.

FDA places clinical hold of RP-A501 for the treatment of patients with Danon disease due to a patient death. Click here to ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.

Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of ...

Rocket Pharmaceuticals shares plummeted 60% Tuesday after the firm said it halted a study of a gene-therapy drug for Danon ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

While vepdegestrant outperformed a standard treatment option in a subtype of patients, the benefit on progression-free ...