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A Devastating Diagnosis, a Viral Video, and a Clinical Trial
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
Diana Kwon | May 1, 2018
When the O’Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.
Families of Children with Rare Diseases Fuel Gene Therapy Research
Families of Children with Rare Diseases Fuel Gene Therapy Research
The Scientist Staff | May 1, 2018
Crowdfunding can power investigations into diseases that would otherwise receive little attention.
How Orphan Drugs Became a Highly Profitable Industry
How Orphan Drugs Became a Highly Profitable Industry
Diana Kwon | May 1, 2018
Government incentives, advances in technology, and an army of patient advocates have spun a successful market—but abuses of the system and exorbitant prices could cause a backlash.
Exome Sequencing Helps Crack Rare Disease Diagnosis
Exome Sequencing Helps Crack Rare Disease Diagnosis
Amanda B. Keener | May 1, 2018
Clinical analyses of patients’ gene sequences are helping to provide answers where none were available before.
Infographic: Piecing the Cholesterol Puzzle
Infographic: Piecing the Cholesterol Puzzle
Diana Kwon | May 1, 2018
How a rare disease led to an understanding of the basics of cholesterol regulation.
Infographic: The Rubber-Hand Illusion
Infographic: The Rubber-Hand Illusion
Roman Liepelt and Jack Brooks | Apr 30, 2017
Simultaneous stroking of one's real hand and a fake limb can lead that person to embody the fake on as their own.
Infographic: Proteasome Basics
Infographic: Proteasome Basics
John Hines and Craig M. Crews | Apr 30, 2017
The structure and function of the cell's protein-degrading machine
Infographic: Watching Evolution in Real Time
Infographic: Watching Evolution in Real Time
Jef Akst | Apr 30, 2017
Guppies transplanted between different communities in Trinidadian streams evolved in response to changes in predation threat in just a few generations.
The Living Set
The Living Set
Wim Hordijk | Jun 1, 2015
Mathematical and computational approaches are making strides in understanding how life might have emerged and organized itself from the basic chemistry of early Earth.