"In 2018, our family welcomed our youngest daughter, Raelin, into the world. From the moment she was born,...
For most of his 10 years, Jacob has been a puzzle. Born prematurely, he faced challenges from the start....
"I was born with a rare disease without even knowing it," explains Fanny Z., a 14-year member of the Society...
Lucas was the youngest of four children. From the start, he stood out—not just as the baby of the family, but...
An Update on Allievex Dearest Sanfilippo Community Members and Allies, It is with deep...
Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel gene...
