Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...

This is read by an automated voice. Please report any issues or inconsistencies here. Health Secretary Robert F. Kennedy Jr. continues to falsely claim that vaccines cause autism despite overwhelming ...

An international research team reveals new molecular mechanisms associated with pathogenic mutations in the protein ...

This work not only identified the genetic cause of RP but also further highlights the role that noncoding genes can have in genetic disorders.

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...

Researchers have identified specific genes containing rare mutations that significantly increase the likelihood of developing attention deficit hyperactivity disorder. These findings, published in the ...

Wounded and elderly patients are being evacuated by train from embattled east Ukraine - Copyright AFP Ishara S. KODIKARA Wounded and elderly patients are being ...

Retinitis pigmentosa (RP) is a genetic eye disorder affecting around one in 5,000 people worldwide. It typically begins with ...

Researchers have understood for decades that some forms of deafness are inherited, yet until now, few of those genetic clues have led the way to therapy. That is beginning to change. An international ...

Researchers from Radboud university medical center and University of Basel have discovered new genetic causes of inherited ...

Patients with PFIC develop liver failure as a result of a buildup of a digestive fluid known as bile. When you purchase through links on our site, we may earn an affiliate commission. Here’s how it ...