Rare diseases
Study sheds light on treatment for rare genetic disorder
Aaron Hoskins鈥� lab partnered with a drug company to understand how RNA-targeting drugs work on spinal muscular atrophy, a disorder resulting from errors in production of a protein related to muscle movement.
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.