Nathalie Guffon
9PUBLICATIONS
3CO-AUTHORS
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Publications (9)
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|Dec 06, 2025
Clinical outcomes of exclusive enzyme therapy (laronidase) in a cohort of patients with mucopolysaccharidosis type I.Nathalie Guffon, Magali Pettazzoni, Nicolas Pangaud
|May 07, 2025
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
|Oct 09, 2024
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
|Feb 28, 2023
Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis: A phase 2, open label, multicenter study.Nathalie Guffon, Vassiliki Konstantopoulou, Julia B Hennermann
|Dec 23, 2022
Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study.Nathalie Guffon, Delphine Genevaz, Didier Lacombe
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