Evidence map›Paper›PMID 35717448›Full record

ArticleThe Journal of pharmacology and experimental therapeutics2022

Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.

N Matthew Ellinwood, Bethann N Valentine, Andrew S Hess, Jackie K Jens, Elizabeth M Snella, Maryam Jamil, Shannon J Hostetter, Nicholas D Jeffery, Jodi D Smith, Suzanne T Millman and 27 more

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In one paragraph

Article in The Journal of pharmacology and experimental therapeutics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.6field-weighted citation impact, top 34% of its field
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

5 citing papers in PubMed, 5 citations in OpenAlex.

  1. Trial
  2. Article
  3. Article
  4. Article
  5. 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

37 authors at 1 institution in 1 country.

N Matthew EllinwoodDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.) matthew@mpssociety.org domainbiotechnologies@gmail.com.
Bethann N ValentineDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Andrew S HessDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Jackie K JensDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Elizabeth M SnellaDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Maryam JamilDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Shannon J HostetterDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Nicholas D JefferyDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Jodi D SmithDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Suzanne T MillmanDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Rebecca L ParsonsDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Mark T ButtDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Sundeep ChandraDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Martin T EgelandDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Ana B AssisDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Hemanth R NelvagalDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Jonathan D CooperDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Igor NestrasilDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Bryon A MuellerDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Rene LabounekDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Amy PaulsonDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Heather PrillDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Xiao Ying LiuDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Huiyu ZhouDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Roger LawrenceDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Brett E CrawfordDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Anita GroverDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Ganesh CheralaDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Andrew C MeltonDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Anu CherukuriDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Brian R VuillemenotDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Jill C M WaitDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Charles A O'NeillDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Jason PinkstaffDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Joseph KovalchinDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Eric ZanelliDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.).
Emma McCullaghDepartments of Animal Science (N.M.E., B.N.V., A.S.H., J.K.J., E.M.S., M.J.), Veterinary Clinical Science (N.M.E., N.D.J.), Veterinary Pathology (S.J.H., J.D.S.), Veterinary Diagnostics and Production Animal Medicine (S.T.M., R.L.P.), and Biomedical Science (S.T.M.), Iowa State University, Ames, Iowa; StageBio, Frederick, Maryland (M.T.B.); BioMarin Pharmaceutical Inc., Novato, California (S.C., H.P., X.Y.L., H.Z., R.L., B.E.C., A.G., G.C., A.C.M., A.C., B.R.V., J.C.M.W., C.A.O., J.P., E.M.); The Lundquist Institute (formerly Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, California (M.T.E., A.B.A., H.R.N., J.D.C.); Department of Pediatrics, Washington University in St Louis, St Louis, Missouri (H.R.N., J.D.C.); Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota (I.N., B.A.M., R.L., A.P.); and Allievex Corporation, Marblehead, Massachusetts (J.K., E.Z.) matthew@mpssociety.org domainbiotechnologies@gmail.com.
The Lundquist Institute · US

Funding

Referral Ctr-Animal models of human genetic diseaseP40OD010939 · OD · UNIVERSITY OF PENNSYLVANIA · PI CASAL, MARGRET L · 2012 to 2023
$7.9M
NIH HHS P40 OD010939
6 · The paper itself

Abstract

Mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome B; OMIM #252920) is a lethal, pediatric, neuropathic, autosomal recessive, and lysosomal storage disease with no approved therapy. Patients are deficient in the activity of N-acetyl-alpha-glucosaminidase (NAGLU; EC 3.2.150), necessary for normal lysosomal degradation of the glycosaminoglycan heparan sulfate (HS). Tralesinidase alfa (TA), a fusion protein comprised of recombinant human NAGLU and a modified human insulin-like growth factor 2, is in development as an enzyme replacement therapy that is administered via intracerebroventricular (ICV) infusion, thus circumventing the blood brain barrier. Previous studies have confirmed ICV infusion results in widespread distribution of TA throughout the brains of mice and nonhuman primates. We assessed the long-term tolerability, pharmacology, and clinical efficacy of TA in a canine model of MPS IIIB over a 20-month study. Long-term administration of TA was well tolerated as compared with administration of vehicle. TA was widely distributed across brain regions, which was confirmed in a follow-up 8-week pharmacokinetic/pharmacodynamic study. MPS IIIB dogs treated for up to 20 months had near-normal levels of HS and nonreducing ends of HS in cerebrospinal fluid and central nervous system (CNS) tissues. TA-treated MPS IIIB dogs performed better on cognitive tests and had improved CNS pathology and decreased cerebellar volume loss relative to vehicle-treated MPS IIIB dogs. These findings demonstrate the ability of TA to prevent or limit the biochemical, pathologic, and cognitive manifestations of canine MPS IIIB disease, thus providing support of its potential long-term tolerability and efficacy in MPS IIIB subjects. SIGNIFICANCE STATEMENT: This work illustrates the efficacy and tolerability of tralesinidase alfa as a potential therapeutic for patients with mucopolysaccharidosis type IIIB (MPS IIIB) by documenting that administration to the central nervous system of MPS IIIB dogs prevents the accumulation of disease-associated glycosaminoglycans in lysosomes, hepatomegaly, cerebellar atrophy, and cognitive decline.

Indexed as

Mucopolysaccharidosis IIIAnimalsBrainChildDisease Models, AnimalDogsEnzyme Replacement TherapyGlycosaminoglycansHeparan SulfateHumansGlycosaminoglycansHeparan Sulfate

Identifiers

PMID35717448
PMCPMC9426762
OpenAlexW4283121428

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