HSPLS site
Login
My List - 0
Help
Search
My Account
Databases
HI Newspaper
eBooks/Audiobooks
Learning
PC Reservation
Reading Program
Basic
Advanced
Power
History
Search:
Title Browse
Author Browse
Subject Browse
Best Seller Browse
Music Title Browse
Video/DVD Title Browse
Journal/Newspaper Title Browse
Serial Title Browse
Series Browse (includes Bestseller List)
General Keyword
Title Keyword
Author Keyword
Subject Keyword
Name Keyword
Series Keyword
Score Title Browse
Talking Book Title Browse
Awards Note Browse
Bib No.
Barcode
Refine Search
> You're searching:
HAWAII STATE PUBLIC LIBRARY SYSTEM
Item Information
Holdings
Summary
More Content
More by this author
Marcus, Amy Dockser, author.
Subjects
Niemann-Pick diseases -- Treatment.
Genetic disorders in children -- Patients -- Family relationships.
Parents of children with disabilities.
Rare diseases -- Patients.
Browse Catalog
by author:
Marcus, Amy Dockser, author.
by title:
We the scientists : ...
MARC Display
We the scientists : how a daring team of parents and doctors forged a new path for medicine / Amy Dockser Marcus.
by
Marcus, Amy Dockser, author.
New York : Riverhead Books, 2023.
Subjects
Niemann-Pick diseases -- Treatment.
Genetic disorders in children -- Patients -- Family relationships.
Parents of children with disabilities.
Rare diseases -- Patients.
ISBN:
9780399576133 (hardcover) :
0399576134 (hardcover) :
Description:
xxx, 222 pages ; 22 cm
Edition:
First edition.
Contents:
Introduction: We the scientists -- The here and now -- A different fear -- The fishing expedition -- The catalysts -- The opera singer -- The broad jump -- New hit -- Reverberations -- Persuasion -- The greater good -- Complications -- The community's drug -- Lilies and doves -- The cathedral of science.
Requests:
0
Summary:
"For more than half a century, medical advances have been driven by investigators launching experiments inside labs. Science is often conducted in isolation and geared toward the long view. This is the story of a group of people who tried to force the lab doors open: parents whose children had been diagnosed with a rare and fatal genetic condition known as Niemann-Pick disease type C. The disease prevents cells from processing cholesterol, which leads to the progressive loss of the brain's and the body's ability to function. Recognizing that there would never be a treatment in time to save their children if things stayed the same, the parents set up a collaboration with researchers and doctors in search of a cure. Reconciling different views of science took work. The parents, doctors, and researchers didn't always agree--among themselves or with each other. But together they endeavored to accelerate the development of new drugs. The parents became citizen scientists, identifying promising new treatments and helping devise experiments. They recorded data about the children and co-authored scientific papers sharing findings. They engaged directly with the FDA at each step of the drug approval process. Along the way, they advanced the radical idea that science must belong to us all. Amy Dockser Marcus shows what happens when a community joins forces with doctors and researchers to try to save children's lives. Their extraordinary social experiment reveals new pathways for treating disease and conducting research. Science may be forever changed."-- Provided by publisher.
Copy/Holding information
Location
Collection
Call No.
Status
Hawaii State Library
Business, Science & Technology
618.92004 Ma
Checked In
Add Copy to MyList
Horizon Information Portal 3.25_9884
© 2001-2013
SirsiDynix
All rights reserved.