Children's Wisconsin Becomes First U.S. Hospital to Administer AVLAYAH, a Blood-Brain Barrier-Crossing Enzyme Replacement Therapy for Hunter Syndrome
核心洞察
Children's Wisconsin (搜索) is the first hospital in the United States to administer AVLAYAH (搜索) following FDA approval, marking a milestone in Hunter syndrome (搜索) treatment.
AVLAYAH (搜索) is the first enzyme replacement therapy engineered to cross the blood-brain barrier, offering potential to prevent and reverse the neuroregressive course of the disease.
Six-year-old Roran Jaskulski is among the first patients in the country receiving the weekly six-hour infusion therapy, with three patients now being treated at Children's Wisconsin (搜索).
Children's Wisconsin (搜索) has become the first hospital in the United States to administer AVLAYAH (搜索), a newly FDA-approved enzyme replacement therapy for Hunter syndrome (搜索), representing what clinicians are calling a revolutionary advance in the management of this rare and devastating genetic disorder.
The milestone was reached under the leadership of Dr. Michael Finkel at Children's Wisconsin (搜索), where 6-year-old Roran Jaskulski of New Berlin is among the first patients in the country to receive the breakthrough treatment. For the past few months, Roran has spent six hours every Friday receiving his infusion at the hospital.
"It's a pretty revolutionary step in Hunter Syndrome (搜索) management," said Dr. Michael Finkel of Children's Wisconsin (搜索).
A Disease with Profound Unmet Need
Hunter syndrome (搜索), also known as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which the body cannot break down certain sugars. This metabolic defect leads to a progressive buildup that damages organs and impairs both physical and mental development. The life expectancy for affected individuals is often between 10 and 20 years old.
Roran was diagnosed two years ago. His mother, Kylie Jaskulski, recalled the devastating guidance the family initially received: "We were just told to take him home and love him and just enjoy the time that we had with him."
Crossing the Blood-Brain Barrier
What distinguishes AVLAYAH (搜索) from previous therapies is its ability to cross the blood-brain barrier — a long-standing challenge in the treatment of lysosomal storage disorders with central nervous system involvement. This is the first enzyme replacement therapy engineered with this capability for Hunter syndrome (搜索).
"Patients that have Hunter Syndrome (搜索), we can actually hopefully prevent and reverse that neuroregressive course," Dr. Finkel explained.
Since Roran's diagnosis, his mother worked closely with the team at Children's Wisconsin (搜索) to secure access to the therapy. Her determination, combined with the hospital's readiness to deliver the treatment, positioned Roran among the earliest recipients in the nation.
Treatment Regimen and Outlook
AVLAYAH (搜索) is administered as a long-term treatment intended to be given for a lifetime. At Children's Wisconsin (搜索), three patients are now receiving the therapy. The Jaskulski family is approaching the future with cautious optimism.
"The goal is to just hope that it gives us more time with him," Kylie Jaskulski said.
The successful administration of AVLAYAH (搜索) at Children's Wisconsin (搜索) signals a meaningful step forward for families facing a diagnosis that has historically offered few therapeutic options, particularly for addressing the neurological dimensions of the disease.