A two-day meeting aimed at raising awareness among policymakers and the public about rare diseases, scheduled for the end of ...

PUMCH-GENESIS currently can flag possible rare genetic diseases or complex neurodevelopmental conditions promptly - Anadolu ...

It’s a race against a single genetic marker -- and musician turned biotech start-up CEO Casey McPherson says the stakes for ...

In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...

When symptoms such as "noticing significant developmental delays in mobility, language, and social interaction since age two" ...

Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, ...

Based on China's accumulated rare disease knowledge and genetic data from its population, PUMCH-GENESIS is the world's first ...

Researchers from the Mitochondrial Medicine Program at Children's Hospital of Philadelphia (CHOP) have better characterized a spectrum of what were classically considered discrete mitochondrial DNA ...

In a global study led by Dr Brian Chung Hong-yin (left), the HKUMed research team identifies a novel gene, DDX39B, for a rare ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Susan Ng and Justine Moss speak with Shukor Yusof, Founder, Endau Analytics, about the need for some airlines to implement ...

New Delhi, Feb 21 (PTI) Government think tank NITI Aayog has formed a committee to create a roadmap aimed at transforming ...