Evidence map›Paper›PMID 41480517›Full record

ArticleJIMD reports2026

Efzimfotase Alfa Improves Respiratory Capacity in Muscle Tissue From a Mouse Model of HPP.

Denise Devore, Juan Ruanova, Walter Voegtli, Derek Dunn, John Decker, Maurizio Mazzantini, Vincenzo De Tata, Maria Concetta Scavuzzo, Francesco Conti, Anna Petryk and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in JIMD reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. Article
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

11 authors.

Denise DevorePharmacology, Alexion, AstraZeneca Rare Disease Boston Massachusetts USA.ORCID https://orcid.org/0009-0005-8219-3233
Juan RuanovaIn Vitro and In Vivo Pharmacology, Alexion, AstraZeneca Rare Disease Boston Massachusetts USA.ORCID https://orcid.org/0009-0000-1269-4650
Walter VoegtliDiscovery Research, Alexion, AstraZeneca Rare Disease Boston Massachusetts USA.
Derek DunnClinical Development, Alexion, AstraZeneca Rare Disease Boston Massachusetts USA.
John DeckerMeTa+ Global SciComm, Alexion, AstraZeneca Rare Disease Boston Massachusetts USA.
Maurizio MazzantiniRheumatology Unit, Department of Clinical and Experimental Medicine University of Pisa Pisa Italy.ORCID https://orcid.org/0000-0003-3741-5990
Vincenzo De TataDepartment of Translational Research and New Technologies in Medicine and Surgery University of Pisa Pisa Italy.ORCID https://orcid.org/0000-0001-8301-4800
Maria Concetta ScavuzzoDepartment of Translational Research and New Technologies in Medicine and Surgery University of Pisa Pisa Italy.ORCID https://orcid.org/0000-0002-4183-9393
Francesco ContiDepartment of Clinical and Molecular Medicine Sapienza University of Rome Rome Italy.ORCID https://orcid.org/0000-0002-9446-6594
Anna PetrykGlobal Medical Affairs, Alexion, AstraZeneca Rare Disease Boston Massachusetts USA.ORCID https://orcid.org/0000-0001-8017-0170
Maria Luisa BrandiFirmoLab, F.I.R.M.O. Italian Foundation for the Research on Bone Diseases Florence Italy.ORCID https://orcid.org/0000-0002-8741-0592

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hypophosphatasia (HPP) is an inherited metabolic disease caused by deficient tissue-nonspecific alkaline phosphatase (ALP) activity and characterized by skeletal and nonskeletal symptoms, including muscle weakness and fatigue. We hypothesized that mitochondrial respiration is impaired in muscle in HPP, independent of skeletal manifestations, and that the second-generation ALP enzyme replacement therapy (ERT) efzimfotase alfa improves respiration. Akp2GW

Indexed as

efzimfotase alfaenzyme replacement therapyhypophosphatasiamitochondrial dysfunctionmuscle weaknessultrastructural studies

Identifiers

PMID41480517
PMCPMC12755057

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.