Publications by authors named "P Harmatz"

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An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.
Joseph Muenzer, Barbara K Burton, Paul Harmatz, Deepa S Rajan, Simon A Jones

N Engl J Med· January 2026

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Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey.
Joseph Muenzer, Jaco Botha, Hernan Amartino, Roberto Giugliani, Paul Harmatz

Mol Genet Metab· December 2025

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Liver iron susceptometry is different in patients with transfusion dependent thalassemia and sickle cell disease compared with hemochromatosis.
Roland Fischer, Marcela G Weyhmiller, Ellen B Fung, Zahra Pakbaz, Doug N Paulson, Paul R Harmatz

Biochem Biophys Rep· December 2025

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Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Eugen Mengel, Rosalia M Da Riol, Mireia Del Toro, Federica Deodato, Matthias Gautschi, Paul Harmatz

Mol Genet Metab· August 2025

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Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey.
Joseph Muenzer, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Shuan-Pei Lin

Orphanet J Rare Dis· July 2025

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