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Rare diseases

New gene new strides in gangliosidosis
Journal News

New gene new strides in gangliosidosis

June 11, 2024

A gene that decreases disease progression in mice provides a new direction for human therapy.

How a gene spurs tooth development
Journal News

How a gene spurs tooth development

May 7, 2024

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
Journal News

A tiny genetic tweak with big heart health implications

Jan. 2, 2024

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?
News

CRISPR and other new technologies open doors, but which diseases get prioritized?

Dec. 17, 2023

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
Health Observance

Understanding the cause of childhood Alzheimer's

Nov. 16, 2023

Today is World Sanfilippo Awareness Day.

Today is International 15q Day
Health Observance

Today is International 15q Day

Nov. 15, 2023

Take some time to read up on the three conditions that constitute this class of neurodevelopmental disorders.

Unraveling amyloidosis, a misunderstood disorder
Health Observance

Unraveling amyloidosis, a misunderstood disorder

Oct. 26, 2023

Today is World Amyloidosis Day.

From genes to hope
Health Observance

From genes to hope

Oct. 19, 2023

Today is Global Niemann鈥揚ick Disease Awareness Day.

Shedding light on Usher syndrome
Health Observance

Shedding light on Usher syndrome

Sept. 16, 2023

On this awareness day, learn about the most common cause of genetic deaf鈥揵lindness and those it affects.