Suchergebnisse - "Pichard, Samia"

PubPharm (61)

1

Effects of miglustat therapy on neurological disorder and survival in early- infantile Niemann-Pick disease type C: a national French retrospective study

enthalten in: ResearchSquare.com | 2023

von Freihuber, C. | Dahmani-Rabehi, B. | Brassier, A. | +11

2

Natural history of GM1 gangliosidosis-Retrospective cohort study of 61 French patients from 1998 to 2019

enthalten in: Journal of inherited metabolic disease | 2023

von Laur, D. | Pichard, S. | Bekri, S. | +7

3

Long-term follow-up of 64 children with classical infantile-onset Pompe disease since 2004 : A French real-life observational study

enthalten in: European journal of neurology | 2023

von Tardieu, M. | Cudejko, C. | Cano, A. | +32

4

Motor outcomes in patients with infantile and juvenile Pompe disease : Lessons from neurophysiological findings

enthalten in: Molecular genetics and metabolism | 2023

von Brassier, A. | Pichard, S. | Schiff, M. | +14

5

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C : a national French retrospective study

enthalten in: Orphanet journal of rare diseases | 2023

von Freihuber, C. | Dahmani-Rabehi, B. | Brassier, A. | +11

6

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study

enthalten in: Orphanet journal of rare diseases | 2023

von Freihuber, C. | Dahmani-Rabehi, B. | Brassier, A. | +11

7

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study

enthalten in: Orphanet journal of rare diseases | 2023

von Freihuber, C. | Dahmani-Rabehi, B. | Brassier, A. | +11

8

Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H : a retrospective study of 51 patients

enthalten in: Bone marrow transplantation | 2023

von Gardin, A. | Castelle, M. | Pichard, S. | +32

9

Individual and Family Determinants for Quality of Life in Parents of Children with Inborn Errors of Metabolism Requiring a Restricted Diet : A Multilevel Analysis Approach

enthalten in: The Journal of pediatrics | 2023

von Ouattara, A. | Resseguier, N. | Cano, A. | +26

10

Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study : The 6-month primary analysis report

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2023

von Kishnani, P. | Kronn, D. | Brassier, A. | +16

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