The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD),…
Articles by Margarida Maia, PhD
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD), a…
For boys who start corticosteroids to treat Duchenne muscular dystrophy (DMD), being taller is linked to slower growth, while…
Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of…
Sarepta Therapeutics is acquiring from Arrowhead Pharmaceuticals the exclusive global rights to develop ARO-DUX4 and ARO-DM1, two RNA…
A woman who developed heart failure due to Duchenne muscular dystrophy (DMD) was diagnosed and treated before she got…
Specific genetic mutations in Duchenne muscular dystrophy (DMD) influence how long patients retain the ability to walk, even when treated…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that…
AOC 1020, an investigational treatment by Avidity Biosciences that’s now called delpacibart braxlosiran, or del-brax, helped adults with…
An engineered transfer RNA (tRNA) molecule developed by hC Bioscience could be a fix for the protein that’s lacking…