MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the first patient has been dosed in ...
Ultragenyx reported third quarter revenue of $160 million, with Crysvita contributing $112 million (North America $57 million, Latin America and Turkey $47 million, Europe $8 million). Dojolvi ...
The FDA approved the drug for Barth syndrome despite findings by its data reviewers that the treatment, while safe, was no ...
In the field of paediatrics, the concept of normal (ie, typical)—in contrast to different, special, deviant, delayed, or ...
The global population is ageing and this demographic shift has profound effects on haemostasis, notably a progressive tilt ...
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