Browsing tag:

Eteplirsen


Eteplirsen CAS 1173755-55-9

The U.S. Food and Drug Administration approved Eteplirsen CAS# 1173755-55-9 (marketed as Exondys 51) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the[…]

Read More

Recent doubt from the FDA regarding Eteplirsen has caused considerable concern for Sarepta Therapeutics Inc. The pending NDA for Eteplirsen has been effectively put on hold by the FDA, based on concerns regarding Phase 2b study results. Patients suffering from Duchenne muscular dystrophy {DMD} are in dire need of effectual, safe and tolerable treatments to[…]

Read More

As a genetic disorder characterized by progressive muscle degeneration and weakness, Duchenne muscular dystrophy, or DMD is a virulent form of muscular dystrophy. The absence of dystrophin, which is a protein that helps to keep muscle cells intact, is the cause of this genetic condition. Duchenne muscular dystrophy is one of the nine types of[…]

Read More