Showing results (1-10 of 101) with videos related to

Sort By:
Pageof 11
Nephrologie & Therapeutique|March 22, 2007
[Enzyme replacement therapy of Fabry's disease: the French experience]Nathalie Guffon
Presse Medicale (Paris, France : 1983)|June 5, 2007
[Overview of enzyme replacement therapy in mucopolysaccharidosis]Alain Fouilhoux, Nathalie Guffon
Paediatric Drugs|January 10, 2016
Long-Term Follow-Up on a Cohort Temporary Utilization Authorization (ATU) Survey of Patients Treated with Pheburane (Sodium Phenylbutyrate) Taste-Masked GranulesYves Kibleur, Nathalie Guffon
JIMD Reports|September 10, 2019
A rare late progression form of Sly syndrome mucopolysaccharidosisNathalie Guffon, Roseline Froissart, Alain Fouilhoux
Journal of Inherited Metabolic Disease|August 2, 2023
Fifty years of research on mitochondrial fatty acid oxidation disorders: The remaining challengesChristine Vianey-Saban, Nathalie Guffon, Alain Fouilhoux, et al.
JIMD Reports|June 24, 2017
Open-Label Single-Sequence Crossover Study Evaluating Pharmacokinetics, Efficacy, and Safety of Once-Daily Dosing of Nitisinone in Patients with Hereditary Tyrosinemia Type 1Nathalie Guffon, Anders Bröijersén, Ingrid Palmgren, et al.
Paediatric Drugs|June 26, 2014
Results from a Nationwide Cohort Temporary Utilization Authorization (ATU) survey of patients in france treated with Pheburane(®) (Sodium Phenylbutyrate) taste-masked granulesYves Kibleur, Dries Dobbelaere, Magalie Barth, et al.
The Journal of Pediatrics|January 27, 2009
Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 yearsNathalie Guffon, Yves Bertrand, Isabelle Forest, et al.
Archives of Disease in Childhood|September 4, 2012
Developing a new formulation of sodium phenylbutyrateNathalie Guffon, Yves Kibleur, William Copalu, et al.
European Journal of Pediatrics|February 12, 2019
Growth impairment and limited range of joint motion in children should raise suspicion of an attenuated form of mucopolysaccharidosis: expert opinionNathalie Guffon, Pierre Journeau, Anaïs Brassier, et al.
Pageof 11