Rare Disease Focus: PKU

Rare Disease Focus: PKU

Biopharma To The Rescue: PKU   The ubiquitous soda can. Who hasn’t seen one? Ever look on the back, at the disturbingly long paragraph of ingredients? The list of ingredients on the back of a can of diet soda are perhaps even more unsettling. Underneath it,...
A Drop Of Gene Therapy

A Drop Of Gene Therapy

A Walk In The Park For Hemophilia? Imagine tripping over your feet during a leisurely stroll down the sidewalk. Ouch! Your knees are scraped below your shorts and blood starts to drip. A quick wipe from a conveniently pocketed napkin and soon enough, you are the proud...
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...