One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.

Gianna Cericola from Philidelphia said the first symptoms she noticed included a burning sensation when she went to the ...

According to Dr. L Swasticharan, Additional DDG with DGHS, if successful, this model could be extended to other rare diseases in India. Study finds how genetic diagnostics of ultra-rare diseases ...

Ultragenyx Pharmaceutical RARE reported fourth-quarter 2024 loss of $1.39 per share, wider than the Zacks Consensus Estimate ...

is undoubtedly the biggest issue for Vimizim and other rare and ultra-rare disease treatments. BioMarin has already offered a confidential 'patient access scheme' (PAS) discount to the £394,680 ...

Total operating expenses for the fourth quarter of 2024 were $287 million, including non-cash stock-based compensation of $40 ...

To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024. The panel ...

BioSkryb Genomics, a Durham company focused on single-cell and ultra-low input multiomics, has introduced a new service called ResolveSEQ MRD, which allows more detailed classification and ...

What are the best and worst aspects of the carnivore diet for your health? And how can you apply these insights to improve your own diet (even if you're not carnivore?) Discuss: The benefits of ...