Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...
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The special reason Terminal Tower will be blue on SaturdayFeb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...
Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only-mode. At the end of the prepared ...
Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...
Angelman Syndrome is a very rare neurogenetic disorder that occurs in approximately one in 15,000 births, caused by the loss of function of a gene ...
The move will provide funds to advance ETX101, a gene therapy the company is developing for SCN1A-positive Dravet syndrome, among other programs.
Rare diseases have become an attractive target for pharmaceutical companies both on the ASX and internationally.
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