Tag:

genetic disease

Acute porphyria: the latest RNA-based clinical trial is a success

Acute porphyria is a group of uncommon diseases that can cause severe, potentially life-threatening attacks of abdominal pain, nausea, vomiting and paralysis. Acute hepatic...

Cystic fibrosis: a more accurate screening will be available

Cystic Fibrosis (CF) is accompanied by chronic obstructive pulmonary disease, pancreatic insufficiency with poor digestion and poor absorption of nutrients, leading to the worsening...

Wanna fight neurodegeneration? SUMO is not the right style

Studying the fundamental aspects of biology can sometimes lead to unexpected findings that directly relate to human disease. In one of the latest examples...

A new drug for autism: unravel, unpack, unsilence.

It has long been presumed that there is a common cause at the genetic, cognitive, and neural levels for autism's characteristic triad of symptoms....