Consensus guidelines for management of ADA-SCID released

Consensus guidelines for management of ADA-SCID released

In light of the advances in the treatment options for ADA deficiency that have occurred since the initial set of guidelines were issued almost a decade ago, the group – comprising clinicians and researchers as well as representatives of patient advocacy groups, the pharmaceutical industry and US government – set out “to review the new…

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Neutropenia a frequent occurrence in ADA-SCID

Neutropenia a frequent occurrence in ADA-SCID

medwireNews: Neutropenia could be a presenting feature of adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID), say investigators after detecting it in more than half of the patients in their chart review.

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Case series of ADA-deficient patients characterised

Case series of ADA-deficient patients characterised

medwireNews: Researchers have characterised the clinical features and outcomes of 13 patients with adenosine deaminase (ADA) deficiency.

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Lymphoproliferative disorders not uncommon in ADA-SCID patients

Lymphoproliferative disorders not uncommon in ADA-SCID patients

medwireNews: There is a “significant” incidence of lymphoma among individuals with adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID), say investigators who identified eight such cases in the literature in addition to their report of a patient with plasmablastic disease.

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Short- and medium-term safety profile of ADA-SCID gene therapy described

Short- and medium-term safety profile of ADA-SCID gene therapy described

medwireNews: Researchers report on the short- and medium-term adverse event (AE) profile of gene therapy with autologous retroviral vector-transduced CD34+ cells in a cohort of 18 patients with adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID).

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Vector-transduced cell tracking feasible in gene therapy-treated ADA-SCID patients

Vector-transduced cell tracking feasible in gene therapy-treated ADA-SCID patients

medwireNews: Japanese researchers have developed a single cell-based method that enables the tracking of vector-transduced cells in adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID) patients treated with stem-cell gene therapy.

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Metabolic, not microbial, pathogenesis likely for ADA-SCID lung disease

Metabolic, not microbial, pathogenesis likely for ADA-SCID lung disease

medwireNews: The underlying cause of the respiratory disease observed in patients with adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID) may be metabolic rather than microbial in nature, research suggests.

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Biodistribution analyses of ADA retroviral vector detailed

Biodistribution analyses of ADA retroviral vector detailed

medwireNews: A report published in Human Gene Therapy Clinical Development describes the preclinical safety studies conducted to support the European market authorisation application for GSK2696273, a gammaretroviral-based gene therapy for adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID).

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Long-term enzyme replacement therapy feasible in ADA-deficient patients

Long-term enzyme replacement therapy feasible in ADA-deficient patients

medwireNews: Enzyme replacement therapy over the long term could be an option for patients with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) for whom curative therapies are unavailable, say US researchers who describe a patient who has remained on treatment for 24 years.

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Cytoreductive conditioning intensity linked to post-gene therapy clonal, TCR diversity

Cytoreductive conditioning intensity linked to post-gene therapy clonal, TCR diversity

medwireNews: In patients with adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID) receiving gammaretroviral gene therapy, the intensity of cytoreductive conditioning could have a bearing on the success of the procedure, in terms of clonal diversity and the T-cell receptor (TCR) repertoire, research indicates.

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