The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy status to NS-089/NCNP-02, NS Pharma’s candidate for…
Articles by Margarida Maia, PhD
Mental health medications, including the stimulant methylphenidate, may help…
Arrowhead Therapeutics has asked for permission to launch a clinical trial of ARO-DUX4, an experimental RNA-based medicine for…
In the U.S., only a limited number of patients will enter Sarepta Therapeutics‘ ENVISION, a Phase 3 study that’s…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on an application to launch a Phase 1…
The U.S. Food and Drug Administration (FDA) has pushed back its decision on SRP-9001 (delandistrogene moxeparvovec), an experimental gene…
IPS Heart has received rare pediatric drug designation for its GIVI-MPC and ISX9-CPC, two experimental single-course stem cell therapies…
A form of congenital muscular dystrophy caused by mutations in a gene called GGPS1 may not always lead to…
Transferring a healthy version of the Large1 gene into adult mice with severe congenital muscular dystrophy restored the function…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ATA-100, a one-time gene therapy being developed…