OpenAlex | RGX-111 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE I (MPS I): INTERIM ANALYSIS OF DATA FROM THE FIRST IN HUMAN STUDY

Rgx 111 Gene Therapy For The Treatment Of Severe Mucopolysaccharidosis Type I Mps I Interim Analysis Of Data From The First In Human Study

Work

Year: 2023

Type: article

Source: Molecular Genetics and Metabolism

Authors Michelle Gilmor, Raymond Wang, Can Fıçıcıoğlu, Roberto Giugliani, Jenna Burke +4 more

Institutions Regenxbio (United States), Children's Hospital of Orange County, Children's Hospital of Philadelphia, National Institute on Population Medical Genetics, Universidade Federal do Rio Grande do Sul

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Cited by: 1

Related to: 10

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Topic: Assisted Reproductive Technology and Twin Pregnancy

Subfield: Pediatrics, Perinatology and Child Health

Field: Medicine

Domain: Health Sciences

Sustainable Development Goal Good health and well-being

Open Access status: closed