Changes in motor function, quality of life, and clinical manifestations in patients with Alpha-mannosidosis treated with Velmanase Alfa: Data from the French Étoile Alpha registry.

Nathalie Guffon, Karine Mention-Mulliez, Francis Gaches, Daniela Garavaglia, Fabrizio Salomone, Bénédicte Héron

Abstract read

In one paragraph

Article in Molecular genetics and metabolism reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0citing papers in PubMed

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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

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3 · Its place in the literature

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0 citing papers in PubMed.

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4 · The record

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5 · Who and what money

Authors and funding

6 authors.

Nathalie GuffonReference Center for Hereditary Metabolic Diseases, Hôpital Femme Mère Enfant - Hospices Civils de Lyon, Lyon, France.

Karine Mention-MulliezReference Center for Hereditary Metabolic Diseases, Hôpital Jeanne de Flandre, CHU Lille, Lille, France.

Francis GachesInternal Medicine, Centre Hospitalier d'Auch, Auch, France.

Daniela GaravagliaBiostatistics, Chiesi Farmaceutici S.p.A., Parma, Italy.

Fabrizio SalomoneMedical Affairs, Chiesi SAS, Bois-Colombes, France.

Bénédicte HéronDepartment of Pediatric Neurology, Reference Center for Lysosomal Diseases, Hôpital Trousseau, Sorbonne University, APHP, Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Alpha-mannosidosis (AM) is a rare lysosomal storage disease with heterogeneous disease manifestations and a continuous spectrum of severity. The only specific treatment for AM is enzyme replacement therapy with velmanase alfa (VA). The focus of this analysis was to assess changes in physician-reported clinical manifestations of AM following VA treatment, using data from Étoile Alpha, a multicenter, non-interventional registry of AM patients treated with VA in France. The cohort included 16 patients (7 females [43.7%]) and 9 males [56.3%]), with a median (range) age at VA initiation and duration of VA treatment of 15.7 (6.1-49.4) and 3.3 (1.1-9.2) years, respectively. Clinical outcomes, including 3-minute stair climb test, 6-minute walk test, forced expiratory volume in 1 second and forced vital capacity, showed numerical improvements from baseline to the last assessment. VA treatment was associated with significant (

Indexed as

Alpha-mannosidosisClinical manifestationsÉtoile alpha registryReal-world evidenceVelmanase alfa

Identifiers

PMID42733529

DOI10.1016/j.ymgmr.2026.101351

PMCPMC13571468

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