Nigel Brasier has competed at various World Disability Billiards and Snooker events around the world [Nigel Brasier] A ...
Full pipelines and rising revenues lead rare disease biotechs to restyle themselves as more mature, global biopharma ...
Among the most famous demonstrations of genetic mutations driving infection outcome is severe combined immunodeficiency (SCID ...
Zycubo received breakthrough therapy, fast track, rare paediatric disease and orphan drug designations from the FDA.
As someone living with a rare disease in Ohio, I know how fragile access to life-saving treatment can be. A bill to help patients with rare or life-threatening illnesses is currently being debated in ...
After their daughter was diagnosed with Sanfilippo syndrome, a Muskego family helped raise millions to support early access ...
It was the priciest biotech startup sale in North Carolina history. Now five years under Bayer, AskBio advances more AAV gene ...
We existed in our own world that day, absorbing information we never wanted to learn. At one point, while I was crying ...
The FDA approved copper histidinate (Zycubo) as the first treatment for Menkes disease in pediatric patients, the agency ...
The U.S. Food and Drug Administration has extended its review for the expanded use of Travere Therapeutics’ drug to treat a ...
The FDA has approved Zycubo (copper histidinate) injection as the first and only treatment of Menkes disease in pediatric patients.
Autoimmune diseases such as multiple sclerosis arise when the immune system turns against the body itself. Yet for most of ...
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