Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...

Race may play a role in the waitlist experience, allograft quality and transplant outcomes of patients with autosomal ...

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene "silencer" that resides in junk DNA is directly sparing people from a devastating ...

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene ...

Creutzfeldt-Jakob disease is an extremely rare and fatal brain-wasting disease that's like a human version of "mad cow." ...

Government of India has provided tax relief on life-saving drugs and medicines used for rare diseases. As per the Ministry of Finance’s response in Rajya Sabha on February 11, 2025, basic customs duty ...

Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...

Millions of people across the UK are suffering from an incurable syndrome that gets worse at night - keeping millions of us ...

A study led by UCL found that individuals with rare diseases were five times more likely to die from Covid-19 than the ...

Nedlands-based biotech company PYC Therapeutics has received approvals to start human trials over a drug candidate for kidney ...

There’s no cure for restless legs syndrome – a distressing disorder that mainly affects sufferers at night – but there are ways to manage and reduce symptoms.

Please provide your email address to receive an email when new articles are posted on . Nephrectomy can be effectively performed in patients with autosomal dominant PKD for various indications ...