Rare diseases
Richard Silverman to speak at ASBMB 2025
Richard Silverman and Melissa Moore are the featured speakers at the ASBMB annual meeting to be held April 12-15 in Chicago.
Becoming a scientific honey bee
At the World Science Forum, a speaker’s call for scientists to go out and “make honey” felt like the answer to a question Katy Brewer had been considering for a long time.
Study sheds light on treatment for rare genetic disorder
Aaron Hoskins’ 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’s 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.