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management change following ngs diagnosis of inborn errors of immunity the australasian experience
Original title: “Management change following NGS diagnosis of inborn errors of immunity: The Australasian experience.”
inborn errors of immunity and hematopoietic cell transplantation recognition and pretransplant management
Original title: “Inborn Errors of Immunity and Hematopoietic Cell Transplantation: Recognition and Pretransplant Management.”
eczema phenotype clustering in inborn errors of immunity with atopy toward better understanding
Original title: “Eczema phenotype clustering in inborn errors of immunity with atopy: toward better understanding.”
evolution of inborn errors of immunity in china discoveries and treatment paradigms
Original title: “Evolution of Inborn Errors of Immunity in China: Discoveries and Treatment Paradigms.”
impact immune monitoring and phenotype assessment of clinical trajectory for patients with inborn errors of immunity from the primary immune deficiency treatment consortium pidtc
Original title: “IMPACT (Immune Monitoring and Phenotype Assessment of Clinical Trajectory) for Patients with Inborn Errors of Immunity from the Primary Immune Deficiency Treatment Consortium (PIDTC).”
interpretation of the classification of human inborn error of immunity 2024 edition
Original title: “[Interpretation of the classification of human inborn error of immunity (2024 edition)].”
emerging trends and research hot spots in inborn error of immunity a bibliometric perspective
Original title: “Emerging trends and research hot spots in inborn error of immunity: A bibliometric perspective.”
lymphoproliferation in inborn errors of immunity mechanisms manifestations and clinical management with a focus on alpid
Original title: “Lymphoproliferation in Inborn Errors of Immunity: Mechanisms, Manifestations and Clinical Management, with a Focus on ALPID.”
Effective gene therapy approaches have been developed for many rare diseases, including <strong>inborn errors of immunity and metabolism, haemoglobinopathies and inherited blindness</strong>. Despite successful pre-clinical and clinical results, these gene therapies are not widely available, primarily for ...
Original title: “Improving access to gene therapy for rare diseases - PMC”