Oak Hill Bio, a new company backed by RA Capital and other investors, will take on Ultragenyx and Ionis in developing an ...

Individualized gene therapies reduce seizures and lead to developmental gains in two boys with severe, treatment-resistant ...

A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment ...

Autosomal dominant Alzheimer's disease (ADAD) is a genetically inherited form of Alzheimer's disease that accounts for only ...

In utero gene therapy uses prenatal genetic screening to identify fetuses with lethal mutations and delivers corrective genes ...

T, Boston Children’s Hospital, and Maine’s Jackson Laboratory announced a collaboration Tuesday to develop gene therapies for ...

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and n-Lorem Foundation, a nonprofit organization, today announced a strategic collaboration and global exclusive license agreement to develop a ...

For decades, we have known IVF for a single promise, which is a positive pregnancy test. Today, that promise has quietly ...

Every person's DNA tells a unique story. To unlock the full potential of genetic research, scientists need tools that reflect ...

The researchers concluded that longitudinal research studies that follow the brain health of South Asian individuals over ...

While Parkinson's disease is more commonly diagnosed in men, growing evidence shows that women may experience different ...

There are more than 30 types of muscular dystrophy and over 7,000 known mutations in the Duchenne gene alone, which has ...