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, there’s a...
New Hope For Spinal Muscular Atrophy

New Hope For Spinal Muscular Atrophy

FIRST THERAPY APPROVED FOR SMA Squeaking by on December 23rd as the last new drug approval of 2016, Biogen’s (Cambridge, MA) Spinraza now provides hope for the thousands of families affected by a debilitating neuromuscular disorder known as spinal muscular atrophy...
The First Three-Parent Baby

The First Three-Parent Baby

BREAKING DOWN THE SCIENCE & SCRUTINY “World’s First Three-Parent Baby” made headlines in publications ranging from Nature to CNN last week. This human interest story is of a baby boy born in Mexico with genetic material from three different parents, achieved by a...
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...