Nathalie Guffon

8PUBLICATIONS
0CO-AUTHORS
Immunogenetics (incl. genetic immunology)Pacific Peoples and disabilityGene and molecular therapyInfant and child healthMedical biochemistry - proteins and peptides (incl. medical proteomics)
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Publications (8)

|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

|Dec 15, 2021
Oral treatment for mucopolysaccharidosis VI: Outcomes of the first phase IIa study with odiparcil.

Nathalie Guffon, Pratima Chowdary, Elisa Leão Teles

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