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

Estimated to affect 0.4 - 0.5 people per million, 1 aHUS is a genetic, ultra-rare disease, which can progressively damage vital organs, such as the kidneys, and can affect both children and adults.

We are a discovery engine and have a pretty good track record for multiple indications including numerous neurological compounds in preclinical studies and even a couple in the clinic for ultra-rare ...

aHUS is an ultra-rare disease caused by abnormalities in the complement system that is estimated to affect only around one person in every two million. It leads to progressive damage to the ...

Estimated to affect 0.4 - 0.5 people per million, 1 aHUS is a genetic, ultra-rare disease, which can progressively damage vital organs, such as the kidneys, and can affect both children and adults. 1 ...

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

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 ...