About ABO-101 (AAV-NAGLU): ABO-101 is Abeonas first-in-human, adeno-associated viral (AAV)-based gene therapy for MPS III
patients ultimately regress to a vegetative state until death, which can occur anywhere between the early teens in the most severe scenarios, to as late as the sixth decade in attenuated forms.
She has the third strain, called Sanfilippo syndrome or MPS III
, which is marked by severe neurological symptoms.
Paul Bean was just five when he was diagnosed with Sanfillipo Syndrome, otherwise known as MPS III
, or Sanfilippo syndrome type A, B, C, and D, and MPS IV A, or Morquio syndrome type A, can present with Hurler-like symptoms, particularly in the early-onset variants.
And while physicians worldwide have diagnosed only 25 to 30 people with MPS VII, one in 70,000 individuals in Israel is born with MPS II, while MPS III
affects one in 24,000 in the Netherlands.