Mucopolysaccharidosis type II (MPSII) is a pediatric lysosomal storage disease caused by deficiencies in the IDS (iduronate-2-sulfatase) gene resultin…

Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

Tříletý Oliver Chu trpěl dědičným Hunterovým syndromem (MPSII), který způsobuje postupné poškození celého těla a mozku. Léčbu se pokusili v Manchesteru úspěšně řešit pomocí unikátní genové terapie.

YouTube video by BBC News

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.Five c...

Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

2026.08.25 　日本新薬は25日、ムコ多糖症Ⅱ型（MPSⅡ、ハンター症候群）治療薬として開発中の「RGX-121」について、提携先のREGENXBIO社（本社：米国）が米国FDAより、臨床試験の実施保留命令（クリ

日本新薬は25日、ムコ多糖症Ⅱ型（MPSⅡ、ハンター症候群）治療薬として開発中の「RGX-121」について、提携先のREGENXBIO社（本社：米国）が米国FDAより、臨床試験の実施保留命令（クリニカル・ホールド）を受けたと発表した。　臨床...

日本新薬は25日、ムコ多糖症Ⅱ型（MPSⅡ、ハンター症候群）治療薬として開発中の「RGX-121」について、提携先のREGENXBIO社（本社：米国）が米国FDAより、臨床試験の実施保留命令（クリニカル・ホールド）を受けたと発表した。　臨床...

Hunter syndrome – or mucopolysaccharidosis type II (MPS II) – is the kind of diagnosis that collapses a family’s sense of time.

**A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy.** Oliver Chu has a rare, inherited condition called Hunter syndrome - or MPSII - which causes progressive damage to the body and brain. Due to a faulty gene, before the treatment Oliver was unable to produce an enzyme crucial for keeping cells healthy. In a world first, medical staff in Manchester have tried to halt the disease by altering Oliver's cells using gene therapy.

日本新薬は25日、ムコ多糖症Ⅱ型（MPSⅡ、ハンター症候群）治療薬として開発中の「RGX-121」について、提携先のREGENXBIO社（本社：米国）が米国FDAより、臨床試験の実施保留命令（クリニカル・ホールド）を受けたと発表した。　臨床...

Three-year-old Oliver Chu has amazed doctors and his parents after he became the world's first patient of a pioneering gene therapy to treat Hunter syndrome. Hunter syndrome - or MPSII - is a rare, inherited condition that causes progressive damage to the body and brain. The effects are sometimes described as a type of childhood dementia. Ollie's dad, Ricky Chu, and the trial's co-lead, Professor Simon Jones, joined Sally and Jon on the BBC Breakfast sofa and described the ''wonderful'' progress Ollie has made in the nine months since the gene therapy. Read more here.

Fergus WalshMedical editor Chu family A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy. Oliver Chu has a rare, inherited condition called Hunter syndrome - or MPSII - which causes progressive damage to the body and brain. In the most severe cases, patients with the disease usually die before the age of 20.

A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy. Oliver Chu has a rare, inherited condition called Hunter syndrome – or MPSII – which causes progressive damage to the body and brain. In the most severe cases, […]

Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.