Rare diseases
New gene new strides in gangliosidosis
A gene that decreases disease progression in mice provides a new direction for human therapy.
How a gene spurs tooth development
University of Iowa researchers find a clue in a rare genetic disorder鈥檚 missing chromosome.
A tiny genetic tweak with big heart health implications
Researchers have discovered that a gene mutation found among members of the Amish community could inform heart disease treatment.
CRISPR and other new technologies open doors, but which diseases get prioritized?
Drug development comes down to money and science. Profits are a factor, but advocacy and incentives can tilt the scale.
Understanding the cause of childhood Alzheimer's
Today is World Sanfilippo Awareness Day.
Today is International 15q Day
Take some time to read up on the three conditions that constitute this class of neurodevelopmental disorders.
Unraveling amyloidosis, a misunderstood disorder
Today is World Amyloidosis Day.
From genes to hope
Today is Global Niemann鈥揚ick Disease Awareness Day.
Shedding light on Usher syndrome
On this awareness day, learn about the most common cause of genetic deaf鈥揵lindness and those it affects.