Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic ...

Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

Rare Disease Day, observed on February 28th, aims to raise awareness about rare diseases and the challenges faced by patients ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

Zevra Therapeutics (ZVRA) announced, on Rare Disease Day, the launch of a new disease state awareness campaign, ‘Learn NPC, Read Between the ...

With issues in therapy delivery and a barren trial landscape, short-term chances for a commercial galactosemia treatment are ...

I envision Chiesi as a proactive leader driving innovation and collaboration within the rare disease community. Our goal is ...

In India, a disease is considered rare if it affects fewer than 1 in 2,500 individuals. The lack of a comprehensive registry ...

For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that ...

Find out how the virtual biotech company Libra Therapeutics leveraged data to accelerate drug development for ...