Suchergebnisse - "Mucopolysaccharidosis"

PubPharm (2.758)

1

Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage

enthalten in: Human gene therapy | 2024

von Catalano, F. | Vlaar, E. | Dammou, Z. | +11

2

MPS II Immunophenotyping : Immunophenotyping of Patients With MPS II Treated With Enzyme Replacement Therapy

enthalten in: ClinicalTrials.gov | 2024

3

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome : A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

enthalten in: ClinicalTrials.gov | 2024

4

Study of Cannabidiol in Sanfilippo Syndrome : Randomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo Syndrome

enthalten in: ClinicalTrials.gov | 2024

5

Ommaya reservoir placement using ultrasound guidance via anterior fontanelle combined with frameless electromagnetic neuronavigation in patients with mucopolysaccharidosis type 2 : Case reports and review of the literature

enthalten in: Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery | 2024

von Mizushima, M. | Kawabori, M. | Yamazaki, K. | +2

6

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 : An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

enthalten in: ClinicalTrials.gov | 2024

7

Further characterization of ARSK-related mucopolysaccharidosis type 10

enthalten in: American journal of medical genetics. Part A | 2024

von Uludağ Alkaya, D. | Taner, H. | Yıldırım, T. | +2

8

Characterization of early markers of disease in the mouse model of mucopolysaccharidosis IIIB

enthalten in: Journal of neurodevelopmental disorders | 2024

von McCullough, K. | Titus, A. | Reardon, K. | +7

9

Characterization of early markers of disease in the mouse model of mucopolysaccharidosis IIIB

enthalten in: Journal of neurodevelopmental disorders | 2024

von McCullough, K. | Titus, A. | Reardon, K. | +7

10

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment : An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment

enthalten in: ClinicalTrials.gov | 2024

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