Suchergebnisse - "Michele Sacchini"

1

Favorable course of previously undiagnosed Methylmalonic Aciduria with Homocystinuria (cblC type) presenting with pulmonary hypertension and aHUS in a young child: a case report

von: Luciano De Simone

2018, in: Italian Journal of Pediatrics

Wird geladen...
2

Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond

von: Antonio Toscano

2023, in: Orphanet Journal of Rare Diseases

Wird geladen...
3

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

von: Rossella Parini

2018, in: Orphanet Journal of Rare Diseases

Wird geladen...
4

Correction: 24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy.

von: Elena Stacy Mazzone

2013, in: PLoS ONE

Wird geladen...
5

24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy.

von: Elena Stacy Mazzone

2013, in: PLoS ONE

Wird geladen...
6

Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.

von: Marika Pane

2014, in: PLoS ONE

Wird geladen...
7

Correction: Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes.

von: Marika Pane

2015, in: PLoS ONE

Wird geladen...
8

Using Cluster Analysis to Overcome the Limits of Traditional Phenotype–Genotype Correlations: The Example of

von: Claudia Dosi

2023, in: Genes

Wird geladen...
9

6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes.

von: Marika Pane

2014, in: PLoS ONE

Wird geladen...
10

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapiesResearch in context

von: Marika Pane

2023, in: EClinicalMedicine

Wird geladen...

Nicht das Richtige dabei?

Schreiben Sie uns!