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ALLMedicine™ Adenosine Deaminase Deficiency Center

Research & Reviews  32 results

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8240285
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...
https://doi.org/10.1111/pde.14597
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...

Selective IgM deficiency: Follow-up and outcome.
https://doi.org/10.1111/pai.13497
Pediatric Allergy and Immunology : Official Publication O... Caka C, Cimen O et. al.

Mar 12th, 2021 - Selective IgM deficiency (sIgMD) is classified under primary immunodeficiencies (PID). This study aimed to define the clinical and immunologic features of sIgMD. We assessed a retrospective medical record of patients who fulfilled the diagnostic c...

Successful engraftment of haploidentical stem cell transplant with post-transplantation...
https://doi.org/10.1111/petr.13954
Pediatric Transplantation REFERENCES; 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...

Outcomes of 3 patients with adenosine deaminase deficiency on long-term enzyme replacem...
https://doi.org/10.1016/j.anai.2020.12.008
Annals of Allergy, Asthma & Immunology : Official Publica... Gans MD, Bernstein L et. al.

Dec 29th, 2020 - Outcomes of 3 patients with adenosine deaminase deficiency on long-term enzyme replacement therapy.|2020|Gans MD,Bernstein L,Shliozberg J,Gavrilova T,Rubinstein A,|

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Drugs  1 results see all →

Clinicaltrials.gov  2 results

Antifolate Effectiveness in Arthritis
https://clinicaltrials.gov/ct2/show/NCT00000395

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
https://clinicaltrials.gov/ct2/show/NCT01279720

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  3 results

EU Regulators Favor 'Bubble Boy' Gene Therapy (Strimvelis)
https://www.medscape.com/viewarticle/861416

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
https://www.medpagetoday.com/genetics/generalgenetics/21301

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
https://www.medpagetoday.com/genetics/generalgenetics/12633

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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