Duchenne muscular dystrophy (DMD)

Aug 20, 2024

Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has recently achieved a significant milestone with the FDA's approval of AMONDYS 45 (casimersen). This antisense oligonucleotide, developed from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, is now approved for treati...

Aug 20, 2024

A rare muscle disorder, Duchenne Muscular Dystrophy (DMD), approximately affects 1 in 3,500 male births globally. In 2023, the total number of prevalent cases of Duchenne Muscular Dystrophy in the 7MM (The US, EU5, and Japan) was around 31,400. The United States encompasses the highest prevalent population of Duche...

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...

Oct 24, 2016

Before approving an investigational drug, safety and effectivity needs to be proven. Something different happened with Sarepta’s Muscular dystrophy drug Eteplirsen, as its luck changed completely when the U.S. Food and Drug Administration on 19th September 2016 approved Sarepta Therapeutics' Exondys 51 (eteplirsen) ...

Dec 22, 2014

Gene Therapy to “fix” Genetic disorders! "We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, "quotes James Watson. This fate and our future is locked in our genes, and Gene therapy is unlocking these doors. Many physicians are predicting that in twent...