About 85 results

ALLMedicine™ Adenosine Deaminase Deficiency Center

Research & Reviews  29 results

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...

Outcomes of 3 patients with adenosine deaminase deficiency on long-term enzyme replacem...
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,|

Urogenital Abnormalities in Adenosine Deaminase Deficiency.
Journal of Clinical Immunology; Pajno R, Pacillo L et. al.

Apr 21st, 2020 - Improved survival in ADA-SCID patients is revealing new aspects of the systemic disorder. Although increasing numbers of reports describe the systemic manifestations of adenosine deaminase deficiency, currently there are no studies in the literatu...

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Drugs  1 results see all →

Clinicaltrials.gov  1 results

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  3 results

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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