Evidence map›Paper›PMID 42496452›Full record

ArticleInternational journal of neonatal screening2026

Combined Intracerebroventricular Enzyme Replacement and Cord Blood Transplantation in Patients with Mucopolysaccharidosis Type II Diagnosed Through Newborn Screening.

Yuki Ueda, Shinsuke Hirabayashi, Masayuki Miura, Satoshi Yamada, Sachiko Nakakubo, Midori Nakajima, Takeru Goto, Jutaro Abe, Yukayo Terashita, Atsushi Manabe and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in International journal of neonatal screening, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Yuki UedaDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.ORCID 0000-0002-6760-6766
Shinsuke HirabayashiDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.ORCID 0000-0001-7961-0968
Masayuki MiuraHokkaido Pharmaceutical Association Public Health Examination Center, Hiragishi 1-jo, 8-chome, Toyohira-Ku, Sapporo 062-0931, Hokkaido, Japan.
Satoshi YamadaDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.
Sachiko NakakuboDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.
Midori NakajimaDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.
Takeru GotoDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.
Jutaro AbeDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.
Yukayo TerashitaDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.ORCID 0000-0003-1700-5337
Atsushi ManabeDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.ORCID 0000-0002-6698-2348
Torayuki OkuyamaDepartment of Genomic Medicine, Saitama Medical University Hospital, Morohongo 38, Moroyamamachi, Iruma-gun, Saitama 350-0451, Saitama, Japan.
Kiyoshi EgawaDepartment of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo 060-8648, Hokkaido, Japan.ORCID 0000-0001-8224-7012

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Enzyme replacement therapy (ERT) for central nervous system symptoms and newborn screening (NBS) is available in Japan for patients with mucopolysaccharidosis type II (MPS II). Of 12 suspected cases identified through the NBS program, 3 patients were diagnosed with neuronopathic MPS II (2 directly from the screened cohort and 1 affected sibling). We reviewed the clinical courses of these patients, who were treated with intracerebroventricular (ICV) ERT using idursulfase beta (Hunterase

Indexed as

cord blood transplantationenzyme replacement therapyintracerebroventricular administrationmucopolysaccharidosis type II

Identifiers

PMID42496452
PMCPMC13398232

What OpenQuestion holds

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LicenceCC BY
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.