ELEVIDYS

Nov 28, 2025

Arrowhead Pharmaceuticals has reached a transformational milestone with the FDA’s approval of REDEMPLO (plozasiran) on November 18, 2025—marking the company’s first FDA-approved medicine and the first-ever FDA-approved siRNA therapeutic for familial chylomicronemia syndrome (FCS) treatment. This historic approval r...

Jun 19, 2024

Duchenne muscular dystrophy (DMD) is a severe genetic disorder primarily affecting young boys between the ages of two and three. As per Delveinsight’s estimates, there were approximately 16K DMD patients in 2023 in the United States, with ˜75% of the patients below the age of 15. This condition leads to progressive...

Jun 30, 2023

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...