Abeona Therapeutics Inc. has announced a significant expansion in access to ZEVASKYN, marking the activation of Children's Hospital of Philadelphia (CHOP) as the sixth Qualified Treatment Center for this groundbreaking…
When baby KJ was diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare metabolic disorder that can trigger life-threatening ammonia buildup, his family faced a narrow path: strict dietary…
Before you read, don't forget to check out Part 1 of our interview with Laura, Nicole's mom. In Part 1, we discuss Nicole's diagnostic journey, what acute lymphoproliferative syndrome (ALPS) is,…
Neuroblastoma has been one of those rare diseases that affects the youth and nobody messes with our future generation! This form of cancer weasels its way and burrows deep in…
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