Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...
Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...
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.
Dr. Eric Levine’s cutting-edge autism research at UConn School of Medicine is reprogramming the donated skin or blood cells ...
South San Francisco, CA, January 30, 2025 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded nearly ...
The governor and Legislature should prioritize services to adults with disabilities and affirm their historical commitment to this special population.
Dr. Roger Hollis from the University of California, Los Angeles, project scientist from the laboratory of Dr. Donald Kohn, a world leader in developing gene replacement therapies using blood stem ...
California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the ...
The data to date, funded by TransformaTx Biotherapeutics, a biotechnology company founded by the Foundation for Angelman Syndrome Therapeutics (FAST), has demonstrated complete symptomatic ...
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