Ahn has studied SON since the early 2000s. After the discovery of ZTTK, she co-founded the ZTTK SON-Shine Foundation in 2021 ...
Baylor College of Medicine has received new funding from the National Institutes of Health for two consortia in the Rare ...
Autism diagnoses rose from 1 in 150 children in 2000 to 1 in 31 in 2022, sparking debate about whether this reflects better detection or a true increase in the disorder.
Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to ...
September is National Sickle Cell Awareness Month in the USA, designated by Congress to help focus attention on the need for ...
There’s one group of people who don’t need convincing that dementia is not an old person’s disease – and that’s the parents ...
A monthslong NBC News data investigation finds that much of the U.S. doesn't have the protection needed to stop the spread of ...
Families of kids with PDCD are urging the FDA to approve DCA, a treatment that could extend their children's lives despite ...
The commercial success of existing lipid-lowering medications highlights the enormous market potential for effective cardiovascular therapies. Read more here.
CAMBRIDGE, MA -- A new study from MIT neuroscientists reveals how rare variants of a gene called ABCA7 may contribute to the development of Alzheimer’s in some of the people who carry it.
JACKSONVILLE, Fla. — A Jacksonville man is walking to end the rare, incurable disease he lives with every day. Tim Nightingale lives with Charcot-Marie-Tooth disease (CMT), which is an inheritable, ...
Copyright 2025 The Associated Press. All Rights Reserved. Copyright 2025 The Associated Press. All Rights Reserved. Since arriving emaciated in Italy from Gaza, Shamm ...