Suchergebnisse - "Gasperini, Serena"

PubPharm (104)

1

Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE) : a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial

enthalten in: EClinicalMedicine | 2024

von Russo, R. | Gasperini, S. | Bubb, G. | +5

2

Long-Term Management of Patients with Mild Urea Cycle Disorders Identified through the Newborn Screening : An Expert Opinion for Clinical Practice

enthalten in: Nutrients | 2023

von Burlina, A. | Gasperini, S. | la Marca, G. | +5

3

Extensive and Persistent Dermal Melanocytosis in a Male Carrier of Mucopolysaccharidosis Type IIIC (Sanfilippo Syndrome) : A Case Report

enthalten in: Children (Basel, Switzerland) | 2023

von Romagnuolo, M. | Moltrasio, C. | Gasperini, S. | +2

4

Antibody Deficiency in Patients with Biallelic KARS1 Mutations

enthalten in: Journal of clinical immunology | 2023

von Saettini, F. | Guerra, F. | Fazio, G. | +21

ErratumIn: J Clin Immunol. 2023 Nov 3;:. - PMID 37921915

5

Parents' experience of the communication process of positivity at newborn screening for metabolic diseases : A qualitative study

enthalten in: Child: care, health and development | 2023

von Bani, M. | Russo, S. | Raggi, E. | +10

6

Correction to : Antibody Deficiency in Patients with Biallelic KARS1 Mutations

enthalten in: Journal of clinical immunology | 2023

von Saettini, F. | Guerra, F. | Fazio, G. | +21

ErratumFor: J Clin Immunol. 2023 Nov;43(8):2115-2125. - PMID 37770806

7

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

enthalten in: Orphanet journal of rare diseases | 2023

von Toscano, A. | Musumeci, O. | Sacchini, M. | +16

8

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

enthalten in: Orphanet journal of rare diseases | 2023

von Toscano, A. | Musumeci, O. | Sacchini, M. | +16

9

Antibody Deficiency in Patients with Biallelic KARS1 Mutations

enthalten in: Journal of clinical immunology | 2023

von Saettini, F. | Guerra, F. | Fazio, G. | +21

10

Finding balance between mature and immature neutrophils : The effects of empagliflozin in GSD-Ib

enthalten in: EJHaem | 2023

von Guerra, F. | Gasperini, S. | Bonanomi, S. | +8

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