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

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 ...

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

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 ...

Local drug developers have sold their rare pediatric disease designation program vouchers for more than $100 million. The FDA ...

Glanders is a rare and potentially deadly disease caused by the bacterium Burkholderia mallei. The World Organization for ...

Ocugen ( NASDAQ: OCGN) disclosed on Tuesday that its gene therapy candidate, OCU410ST, has been granted rare pediatric ...

People with autism spectrum disorder (ASD) had a higher risk of developing Parkinson's disease early in life, a nationwide ...

Prothena has buried birtamimab for the second time. A phase 3 trial of the anti-amyloid antibody in the rare disease AL ...

A Spokane County employee and his family are sending gratitude to his fellow union members, who donated PTO to allow him to keep his job for at least 12 more weeks.