All About Oliver

A Sanfilippo Story by Grammy L…

The Best Birthday Gift

It finally happened! Oliver pre-tested, tested again and again, qualified for the clinical trial dosage of ABO-101 and thanks to Dr. Kevin M. Flanigan (who jumped up, down, through, over, and under hoops to make this happen during the COVID-19 pandemic no less) Oliver checked in to Nationwide Children’s Hospital in Columbus, Ohio.

I knew this was a possibility as Jen and Brian sat on pins and needles waiting for the green light as the trial dosage shipped from the U.K. , arrived in NYC, and was held up in customs. The light remained yellow.

And then it turned green!

Text messages started dinging…

I was shocked, deliriously happy and overcome with love…

And in a private text I said…

I can’t help being Grammy L, the cheerleader for my family…


Sanfilippo Syndrome Type B, also known as mucopolysaccharidosis type IIIB (MPSIIIB), is characterized by the body’s inability to break down large sugar molecules called mucopolysaccharides, or glycosaminoglycans.

In patients with MPSIIIB, gene mutations result in a marked decrease in NAGLU enzyme activity, which leads to an accumulation of heparin sulfate in the brain and other organs, as well as progressive brain atrophy.

Essentially, ABO-101 is a virus that can cross the blood brain barrier after intravenous administration.

Dr. Flanigan is the trial’s principal investigator and Director of the Center for Gene Therapy at Nationwide Children’s Hospital.

“We are especially pleased to see reductions in several key biopotency markers, including the reductions in cerebral spinal fluid, urine and plasma heparan sulfate and normalization of plasma NAGLU enzyme activity at days 7, 14, and 30 post-transfer, added Flanigan, also a professor of pediatrics and neurology at The Ohio State University College of Medicine.” (sanfilipponews.com, courtesy of Jose Marques Lopes, PHD)


Oh, Happy Day!

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