The "Valley of Death" Looms for 8 Kids with a Rare Disease

<p>The pharmaceutical industry righteously calls a theatre in drug growth between simple investigate and clinical trials a Valley of Death. This is when a intensity diagnosis that s worked in mice, monkeys, and a like catapults to a proviso 1 clinical hearing to consider safety. It s rare.
Francis Collins, MD, PhD, executive of a National Institutes of Health, calls this duration where projects go to die. The reason: $.
Matthew Herper writes in that a cost of building a new drug is $4-11 billion, not a $1 billion that Pharma mostly claims. Yet even that $1 billion is unimaginable, generally when we put a face on a singular illness and declare what a family goes by to jump to proviso 1.
Ginger and Hannah. Photo: Dr. Wendy Josephs
For me, that face belongs to 8-year-old Hannah Sames, of Rexford, New York.
Hannah is one of customarily 54 people famous in a universe to have hulk axonal neuropathy, or GAN. It s like ALS (Lou Gehrig s disease) in a child. Today Hannah needs a wal...