Ed Kaye
Management
[Call Starts Abruptly] Successfully achieved in Professor Steve Wilton’s lab. So we could not be happier for today’s approval, which allows EXONDYS 51 and exon skipping drug should be available to patients in the United States. This extraordinary community has supported, encouraged, and inspired our work on creating therapies to treat the underlying cause of Duchenne muscular dystrophy. I would like to acknowledge of all the people living with DMD, and in particular, the 12 boys who participated in our 201, 202 trial, who allowed us to study them for five years and also providing a novel treatment for others, who have been combating this crippling disease. I was speaking to patients as well as caregivers, physicians, and investigators. This approval is a direct result of your commitment and resolve. We could not be prouder of this achievement and we thank you. As many of you know, DMD is a pediatric X-linked recessive neuromuscular disease, caused by mutations in the DMD gene, which prevents the reduction of functional dystrophin protein. Dystrophin plays a vital role in the structure, function, and preservation of muscle cells. The progressive loss of muscle function culminates in respiratory and cardiac complications that result in premature death. Eteplirsen is designed to bind to Exon 51 of dystrophin pre-messenger RNA, resulting in exclusion of this exon during messenger RNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy and patients who have a confirmed mutation of the DMD gene that is amenable to Exon 51 skipping. This indication is approved under accelerated approval, based on an increase in dystrophin and skeletal muscle observed in some patients treated with…