Article

Management of MPS-I using ERT: The Egyptian experience

2023-11-16

Abstract excerpt

<title>Abstract</title><p>Background Mucopolysaccharidosis type I (MPS I) is an autosomal recessive multisystem lysosomal storage disorder. Methods Herein, we report the Egyptian experience of enzyme replacement therapy (ERT) for MPS type I patients and the faced challenges. Thirty-eight MPS-I patients were examined at presentation and throughout ERT to evaluate its effect on different body systems. Clinical and r...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 54ea16b6-61ba-50f9-a3d4-7f5a4ccec42d
DOI: 10.21203/rs.3.rs-3573710/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Management of MPS-I using ERT: The Egyptian experienceDOI 10.21203/rs.3.rs-3573710/v1Read

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare diseaseSemantic proximity Read

A Review Of The Clinical Outcomes In Idursulfase-Treated And Untreated Filipino Patients With Mucopolysaccharidosis Type II: Data From The Local Lysosomal Storage Disease RegistrySemantic proximity Read

Mucopolysaccharidosis I, II, and VI: brief review and guidelines for treatmentSemantic proximity Read

Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-year Clinical Outcome Study of the First Taiwanese CaseSemantic proximity Read

Phase I and II clinical trials for the mucopolysaccharidosesSemantic proximity Read

Treating lysosomal storage disorders: What have we learnt?Semantic proximity Read

The Landscape of Mucopolysaccharidosis in Southern and Eastern European Countries: A Survey from 19 Specialistic Centers.Semantic proximity Read

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and BeyondSemantic proximity Read

Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry.Semantic proximity Read

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy.Semantic proximity Read

The landscape of Mucopolysaccharidosis in Southern and Eastern European countries: a survey from 19 specialistic centersSemantic proximity Read

Updates in Advances in Therapeutic Options for Pulmonary and Sleep Disorders in Mucopolysaccharidosis (MPS) Patients: A Narrative ReviewSemantic proximity Read