David Shortlidge, Age 6 Derry, NH Home and Family David lives with his parents and 4 year old sister. He has two pets at home; a cat named Bodie and a fish named Nemo. Diagnosis David was diagnosed with Duchenne muscular dystrophy when he was 3 years old. DMD is a genetic disorder characterized by . . .
Andrew Nichols, Age 8 Newark, OH Home and Family Andrew lives at home with his younger brother Ethan, and parents Jodi and Mitch. He loves his animals and especially his soon to be service dog, Hero! Diagnosis David was diagnosed with Duchenne muscular dystrophy when he was 3 years old. DMD is a genetic disorder . . .
Caleb David Pendley, Age 14 Oklahoma Home and Family Caleb has two loving parents, Marlene and Noel, along with three siblings; Isaac (17), Micah (11) and Abigail (9). His family recently traveled to the Grand Canyon and they had a great time! Diagnosis Caleb was diagnosed with Duchenne muscular dystrophy at the age of 8. . . .
Trent Tomco, Age 11 Aiken, SC Home and Family Trent is the son of Michelle and Paul Tomco. He has two brothers and two sisters. Diagnosis Trent has Duchenne muscular dystrophy (DMD). DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. DMD is caused by an absence of dystrophin, a protein that . . .
Devin Sundquist, Age 11 Orem, UT Home and Family Devin lives at home with three sisters, his mom and step dad. He loves to play board games and hang out with his family. Diagnosis Devin was diagnosed with Duchenne muscular dystrophy at the age of seven. Duchenne muscular dystrophy (DMD) is a genetic disorder characterized . . .
Background: PTC Therapeutics yesterday announced results from the phase 3 “ACT DMD” trial of ataluren, an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). The trial results showed clinically meaningful benefits for ataluren-treated patients, indicating the drug may slow functional decline in DMD patients, with some patients apparently . . .
Background: Sarepta Therapeutics reported today additional long-term clinical efficacy and safety data from its pivotal phase 2b program of eteplirsen to treat Duchenne muscular dystrophy (DMD). The data demonstrated that over a three-year period of time, trial participants who were treated with eteplirsen experienced a slower rate of decline and were, on average, able to . . .
Background: Sarepta announced today that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for eteplirsen to treat Duchenne muscular dystrophy (DMD). Eteplirsen is an “exon-skipping” drug that targets a section of DNA called exon 51, and may help up to 13 percent of Duchenne muscular dystrophy (DMD) patients. Exon . . .
Following a pre-NDA meeting with the U.S. Food and Drug Administration (FDA), Marathon Pharmaceuticals has announced that it will begin the New Drug Application (NDA) process for deflazacort as a treatment for Duchenne muscular dystrophy (DMD). Marathon expects to submit the NDA in the first quarter of 2016. If the application is approved, the company . . .
Background: On Monday, June 29, BioMarin announced that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen. Both “exon-skipping” drugs target exon 51, and may help up to 13% of Duchenne muscular dystrophy patients. Exon skipping is a treatment strategy in which sections of genetic . . .
