The PLN gene mutation causes heart failure and sudden cardiac death. Experts say West Michigan's Dutch community could be ...

A research team led by Purdue University's W. Andy Tao has discovered a new type of protein modification related to cellular mutation that impairs a crucial enzyme's ability to help drive energy ...

FDA introduced a new "Plausible Mechanism Framework." Under this policy, certain gene therapies and RNA-based treatments ...

India's first national biobank for rare Lysosomal Storage Disorders has been established in Ahmedabad, Gujarat. The government-funded facility aims to enable affordable diagnosis and treatment by ...

VG801 shows encouraging preliminary efficacy in the ongoing Phase 1/2 trial, with consistent functional improvements in best-corrected visual ...

ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today ...

Rare medical events draw intense attention during any mass vaccination campaign. Among COVID vaccine side effects, few ...

IN the vibrant city of Dublin, Ireland, over 2,000 delegates from over 77 countries gathered for the 19th annual European Association ...

In brain tissue from people who died with aFTLD-U – a rare subtype of dementia marked by early behavior change – chromosome ...

A 20-year study has shown that, like photocopying photocopies, cloning doesn't produce perfect copies – and the reason could be that the cloning process itself produces lots of mutations ...

Scientists at Sanford Burnham Prebys Medical Discovery Institute and an international team of collaborators have defined a new genetic disease marked by premature aging and deficits in brain function.

A blood test clarifies which gene variants truly cause congenital heart defects, linking diagnosis to more precise ...