Browsing: Clinical Trials

Rocket Pharmaceuticals sold its rare disease pediatric priority review voucher for $180 million, cash that the company will use to…

A Novartis RNA therapy reduced blood levels of a protein that’s a biological indicator of a particular form of muscular…

Many proteins in the body are known to cause disease but remain stubbornly out of reach for the traditional ways…

For severe cases of the bleeding disorder von Willebrand disease, patients must make frequent visits to treatment centers for infusions…

Artificial intelligence (AI) is transforming how medicines are discovered, but clinical trials are still governed by a structure that has…

Targeted therapies are bringing new treatment options for a wide range of cancers, but drug resistance still looms for these…

The operational structure of the FDA around centers, each governing a particular regulatory or scientific area, dates back more than…

With few treatments available for sickle cell disease, encouraging early clinical data for a Fulcrum Therapeutics drug made it one…

Servier is expanding its scope in neurology to include neuromuscular disorders by acquiring a clinical-stage Edgewise Therapeutics drug that offers…

In the rare blood disorder light chain amyloidosis, standard treatment is a drug combination used for a blood cancer. It’s…