A man who should have developed early-onset Alzheimer's disease due to a genetic mutation is still symptom-free in his 70s.

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

From life-altering genetic conditions to promising new treatments, here's what you need to know about 7 inherited diseases ...

Only around two percent of the human genome codes for proteins, and while those proteins carry out many important functions ...

In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...

In a global study led by Dr Brian Chung Hong-yin (left), the HKUMed research team identifies a novel gene, DDX39B, for a rare ...

Did you see this? Nearly 1 in 10 Americans have asthma. Here's what causes it.

Neurogeneticists have discovered a novel genetic mutation and associated buildup of toxic proteins in the brain -- a type of buildup distinct from amyloid or tau, proteins that have long been the ...

An FDA-approved osteoporosis treatment might nudge a mutated protein back into shape and improve heart function, according to ...

WashU Medicine-led trial evaluating investigational drug from Eli Lilly and Company aims to stop disease before symptoms ...

A groundbreaking study has identified how previously mysterious mutations in non-coding DNA contribute to kidney disease, ...

Passage Bio is outsourcing future analytical testing services in a cost-cutting move that will further cut its workforce, a ...