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Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report

Journal of personalized medicine, 2023-04, Vol.13 (4), p.637

2023

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P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up

Genetics in Medicine Open, 2024, Vol.2, p.100882, Article 100882

2024

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P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I‐II CLINICAL TRIAL OF EX‐VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW‐UP

HemaSphere, 2023-08, Vol.7 (S3), p.e1995859-n/a

2023

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Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome

Science translational medicine, 2024-05, Vol.16 (745), p.eadi8214

2024

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Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107831, Article 107831

2024

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Long-term clinical outcomes of atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107108, Article 107108

2023

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Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107044, Article 107044

2023

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First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S121-S122

2022

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Spontaneous fractures in custom-made porous hydroxyapatite cranioplasty implants: is fragility the only culprit?

Acta neurochirurgica, 2015-03, Vol.157 (3), p.517-523

2015

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Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report

Journal of personalized medicine, 2023, Vol.13 (4)

2023

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Prevalence of Serum Antibody Titers against Core Vaccine Antigens in Italian Cats

Life (Basel, Switzerland), 2023-11, Vol.13 (12), p.2249

2023

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Performance Assessment of Two Commercial Amplification Assays for Direct Detection of Mycobacterium tuberculosis Complex from Respiratory and Extrapulmonary Specimens

Journal of Clinical Microbiology, 2002-11, Vol.40 (11), p.4138-4142

2002

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Comparison of MB/BacT ALERT 3D System with Radiometric BACTEC System and Löwenstein-Jensen Medium for Recovery and Identification of Mycobacteria from Clinical Specimens: a Multicenter Study

Journal of Clinical Microbiology, 2001-02, Vol.39 (2), p.651-657

2001

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A cluster of factor XI-deficient patients due to a new mutation (Ile 436 Lys) in northeastern Italy

European journal of haematology, 2012-03, Vol.88 (3), p.229-236

2012

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Comparative Evaluation of the New Gen-Probe Mycobacterium tuberculosis Amplified Direct Test and the Semiautomated Abbott LCx Mycobacterium tuberculosis Assay for Direct Detection of Mycobacterium tuberculosis Complex in Respiratory and Extrapulmonary Specimens

Journal of Clinical Microbiology, 1998-12, Vol.36 (12), p.3601-3604

1998

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Volatile Anesthetics for Intensive Care Unit Sedation

Critical Care Sedation, 2018, p.103-119

2018

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Comparison of MB/BacT ALERT 3D System with Radiometric BACTEC System and Löwenstein-Jensen Medium for Recovery and Identification of Mycobacteria from Clinical Specimens: a Multicenter Study

Journal of clinical microbiology, 2001-02, Vol.39 (2), p.651-657

2001

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Automatic modeling and authoring of nonlinear interactions between acoustic objects

The Fourth International Workshop on Multidimensional Systems, 2005. NDS 2005, 2005, p.116-122

2005

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An integrated system for the automatic block-wise synthesis of sounds

13th European Signal Processing Conference (EUSIPCO 2005), 2005, p.1-4

2005

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Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report

P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up

P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I‐II CLINICAL TRIAL OF EX‐VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW‐UP

Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome

Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up

Long-term clinical outcomes of atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up

Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use

First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction

Spontaneous fractures in custom-made porous hydroxyapatite cranioplasty implants: is fragility the only culprit?

Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report

Prevalence of Serum Antibody Titers against Core Vaccine Antigens in Italian Cats

Performance Assessment of Two Commercial Amplification Assays for Direct Detection of Mycobacterium tuberculosis Complex from Respiratory and Extrapulmonary Specimens

Comparison of MB/BacT ALERT 3D System with Radiometric BACTEC System and Löwenstein-Jensen Medium for Recovery and Identification of Mycobacteria from Clinical Specimens: a Multicenter Study

A cluster of factor XI-deficient patients due to a new mutation (Ile 436 Lys) in northeastern Italy

Comparative Evaluation of the New Gen-Probe Mycobacterium tuberculosis Amplified Direct Test and the Semiautomated Abbott LCx Mycobacterium tuberculosis Assay for Direct Detection of Mycobacterium tuberculosis Complex in Respiratory and Extrapulmonary Specimens

Volatile Anesthetics for Intensive Care Unit Sedation

Comparison of MB/BacT ALERT 3D System with Radiometric BACTEC System and Löwenstein-Jensen Medium for Recovery and Identification of Mycobacteria from Clinical Specimens: a Multicenter Study

Automatic modeling and authoring of nonlinear interactions between acoustic objects

An integrated system for the automatic block-wise synthesis of sounds

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