The STING protein, known for helping cells fight viral infections by generating inflammation, also appears to function as a ...

The biotech is developing an experimental drug for the most common of a family of disorders all caused by errors in ...

AI and digital health have tremendous potential to improve patient outcomes across healthcare, but in rare disease – where small patient groups, delayed diagnosis, and barriers to access create major ...

Some of the more common and important genetic metabolic disorders include: Lysosomal storage disorders. Lysosomes are spaces inside cells that break down waste products of metabolism. Various ...

In this webinar – ‘Enabling Digitally Enhanced Care for Rare Diseases in Europe: Integrating AI and Connected Care ...

Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease originating from biallelic pathogenic variants in the ARSA gene, mainly affecting young children.

Denali Therapeutics is advancing the fight against neurodegenerative diseases thanks to pioneering work in methods to address the root issues of a host of debilitating conditions.

A research team led by Prof. ZHANG Hong from the Institute of Biophysics of the Chinese Academy of Sciences has uncovered the molecular mechanism by which the key autophagy protein ATG-9 promotes the ...

With the annual cost estimated to be about ₹1 crore for each child requiring treatment for ultra-rare diseases, the Medical ...

The lead asset is DNL310, a recombinant, brain penetrating IDS enzyme molecule targeting Hunter syndrome (MPS II), a rare neurodegenerative lysosomal storage disorder. The company is planning a ...