Exon-Skipping Therapy For DMD

Exon-Skipping Therapy For DMD

The Science Behind Sarepta’s Hotly Debated Antisense Drug Sarepta’s (Cambridge, MA) Duchenne muscular dystrophy drug Exondys 51 crossed the finish line earlier this week, with a conditional stamp of approval by the FDA. This hotly debated regulatory result...
Exon-Skipping Therapy For DMD

The Powered Exoskeleton

THINKING OUTSIDE OF THE BODY Last WEEKLY’s focus on Duchenne muscular dystrophy got us to thinking, what products are available for those with limited mobility? A new type of medical device called a powered exoskeleton certainly caught our eye. Originally...
Deciphering Duchenne Muscular Dystrophy

Deciphering Duchenne Muscular Dystrophy

THE ROOT OF DUCHENNE MUSCULAR DYSTROPHY World Duchenne Awareness Day called attention to Duchenne Muscular Dystrophy (DMD) this past Monday. Affecting one in 3,500 newborn baby boys worldwide, this fatal disease is caused by a mutation in the gene that codes for the...