On Saturday, May 24, the 78th World Health Assembly (WHA) voted to adopt a resolution to establish a global framework to ...

A new rapid blood test for newborns could potentially detect genetic mutations linked to thousands of rare diseases all at ...

U.S. President Donald Trump's trade negotiators are pushing the EU to make unilateral tariff reductions on U.S. goods, saying ...

Whipple’s disease is a rare bacterial illness affecting multiple organs. Early diagnosis and long-term antibiotics are key to ...

Sun Pharmaceutical , India's largest drugmaker by revenue, reported a bigger adjusted quarterly profit on Thursday, driven by ...

The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated ...

OCU410ST has received Rare Pediatric Disease Designation for ABCA4-associated retinopathies, potentially allowing Ocugen to ...

A new, rapid testing method will greatly help the diagnosis of rare diseases in babies and children, according to research ...

Genentech’s short film draws on the experiences of multiple families caring for children with hemophilia A, seeking to ...

Corina and Destiny lost their first child, Ahi o Te Aroha, at six months old. Although genetic testing identified a variant ...

Wilson's new company, Philadelphia-based Gemma Biotherapeutics, plans to start testing gene therapies in Brazil this year.

Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this ...