About 118 results

ALLMedicine™ Adenosine Deaminase Deficiency Center

Research & Reviews  39 results

Balancing early detection of adenosine deaminase deficiency with overdiagnosis risk.
Annals of Allergy, Asthma & Immunology : Official Publica... Revak S, Shaker M et. al.

Dec 5th, 2022 - Balancing early detection of adenosine deaminase deficiency with overdiagnosis risk.|2022|Revak S,Shaker M,Sun D,|diagnosis,genetics,

Newborn tandem mass spectroscopy screening for adenosine deaminase deficiency.
Annals of Allergy, Asthma & Immunology : Official Publica... Hartog N, Hershfield M et. al.

Aug 2nd, 2022 - Newborn screening (NBS) by means of T cell receptor excision circles (TREC) is now universal in the United States, Puerto Rico, and the Navajo Nation as a strategy to identify severe combined immunodeficiency (SCID) in newborns. Owing to the chara...

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
The New England Journal of Medicine; Kohn DB, Booth C et. al.

May 12th, 2021 - Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United Kingdom) with an in...

Multicentric dermatofibrosarcoma protuberans in a child with severe combined immunodefi...
Pediatric Dermatology; Wahjudi TD, Kutzner H et. al.

May 2nd, 2021 - We report the case of a 4-year-old boy, post-human stem cell transplantation for severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency (ADA), who developed multiple dermatofibrosarcoma protuberans (DFSP). We hypothesize a r...

Successful engraftment of haploidentical stem cell transplant with post-transplantation...
Pediatric Transplantation; Medina D, Aristizabal AM et. al.

Dec 30th, 2020 - SCID are characterized by an imbalance in cellular and humoral immunity. Enzyme ADA deficiency represents from 10% to 15% of the SCID. This generates diminished maturation of the cell precursors. Treatments include enzyme replacement therapy, allo...

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Drugs  1 results see all →

Clinicaltrials.gov  2 results

Antifolate Effectiveness in Arthritis

Dec 16th, 2016 - Low-dose methotrexate therapy suppresses autoimmune arthritis in human and animal models. We hypothesize that the effect of methotrexate in the treatment of rheumatoid arthritis is due to the inhibition of aminoimidazole-carboxamide ribotide trans...

Gene Therapy ADA Deficiency

Sep 14th, 2015 - Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.

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News  5 results

Early Win for Gene Therapy in Rare Form of SCID

Dec 21st, 2022 - A gene therapy for a rare type of severe combined immunodeficiency (SCID) was safe and sufficiently restored T-cell immunity in an early trial, results from a phase I/II study showed. At a single center, all 10 infants with Artemis-deficient SCID ...

Gene Therapy Fixes Rare Type of Deadly Immune Deficiency

May 11th, 2021 - A novel gene therapy for severe combined immunodeficiency from adenosine deaminase deficiency (ADA-SCID) restored immune cell counts without death or need for enzyme replacement therapy, researchers reported. Survival without needing to re-initiat...

EU Regulators Favor 'Bubble Boy' Gene Therapy (Strimvelis)

Apr 4th, 2016 - Editor’s note: The European Commission approved the gene therapy Strimvelis (GlaxoSmithKline) on May 27, 2016, to treat children with the very rare disease Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), accordin...

Gene Therapy Benefits Persist in SCID

Jul 21st, 2010 - Action Points Explain to interested patients and parents that gene therapy for severe combined immunodeficiency disease (SCID) was shown to be safe for up to 10 years in a small number of patients with the lethal genetic disease. But caution that ...

Gene Therapy Works for Rare Immune Disorder

Jan 28th, 2009 - Action Points Explain to interested patients that this study found that replacing a defective gene provided improvements in the immune systems of almost all of the children with severe combined immunodeficiency caused by lack of adenosine deaminas...

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