Publications by authors named "N Guffon"

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Clinical outcomes of exclusive enzyme therapy (laronidase) in a cohort of patients with mucopolysaccharidosis type I.
Nathalie Guffon, Magali Pettazzoni, Nicolas Pangaud, Nathalie Reynes, Eliane Le Peillet Feuillet

Orphanet J Rare Dis· December 2025

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Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).
Karolina M Stepien, Barbara K Burton, Michael B Bober, Philippe M Campeau, Carolyn Ellaway, Nathalie Guffon

Orphanet J Rare Dis· October 2025

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Long-Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency.
Maurizio Scarpa, George A Diaz, Roberto Giugliani, Simon A Jones, Eugen Mengel, Nathalie Guffon

J Inherit Metab Dis· September 2025

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Evolution of mobility, pain/discomfort, self-care, and mental health in patients with alpha-mannosidosis: an international caregiver and patient survey.
Karolina M Stepien, Sophie Thomas, Julia B Hennermann, Christina Lampe, Nicole M Muschol, Nathalie Guffon

Orphanet J Rare Dis· May 2025

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Extended long-term efficacy and safety of velmanase alfa treatment up to 12 years in patients with alpha-mannosidosis.
Nathalie Guffon, Line Borgwardt, Anna Tylki-Szymańska, Andrea Ballabeni, Francesca Donà

J Inherit Metab Dis· January 2025

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