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Primary Immune Deficiencies - An OverviewPublished by: Datamonitor Published: Oct. 21, 2002 - 32 Pages Table of ContentsOverview Introduction Primary immune deficiencies are inborn genetic defects in the immune system. It is estimated that at least half a million people worldwide suffer from at least one of these disorders. These patients suffer from recurrent and persistent infections. Gene therapy offers the promise of a potential cure, obliterating the need for antibiotic and immunoglobulin therapy and bone marrow transplants. Scope Examines disease etiology, symptoms and treatment options available to patients Looks at the prevalence and diagnosis of some of the more common and defined conditions and discusses the prognosis for these individuals Assesses unmet needs in the treatment of primary immune deficiencies and future opportunities, particularly in the area of gene therapy Report Highlights Prompt administration of antibiotics at the first signs of infection is vital and those with deficient antibody levels require monthly immunogobulin infusions. Bone marrow transplantation has provided complete remission to some suffering from very severe conditions, such as severe combined immunodeficiency disease (SCID), but global success rates over the past thirty years are only between 60-70%. Gene therapy is beginning to show some promise as a viable treatment alternative. The obvious benefit in curing these debilitating illnesses will have to be balanced with the potential risks posed by this new form of treatment. Although entry into the commercial arena is not expected for another few years, the market and growth for gene therapy products will be unprecedented, since application of this technology will have far-reaching implications for a number of other clinical conditions. Reasons to Purchase Insightful overview of the key features of primary immune deficiencies, including prevalence, diagnosis and symptoms Analyse current treatment options and prognosis for some of the more defined and common disorders Identify unmet needs and learn about future opportunities, especially in gene therapy, which has enormous market potential DRIVERS AND TRENDS Primary immune deficiencies, of which there are approximately 90 different forms, are inborn genetic defects in the immune system. It is estimated that at least half a million people worldwide suffer from at least one of these largely incurable disorders. These patients suffer from recurrent and persistent infections. Bone marrow transplantation has provided complete remission to some suffering from very severe conditions, such as severe combined immunodeficiency disease (SCID), but global success rates over the past thirty years are only between 60-70%. Although still a few years from the first commercial product, gene therapy offers the real promise of a viable treatment alternative, thereby obliterating the need for current treatments including life—long antibiotic and immunoglobulin therapy. INTRODUCTION TO PRIMARY IMMUNE DEFICIENCIES Future challenges and opportunities in the treatment of primary immune deficiencies are assessed. Very recently, calls have been made that some gene therapy clinical trials, which had been suspended due to adverse side-effects, be re-instigated. This highlights the desperate need for a cure for these conditions. A complete overview of primary immune deficiencies is provided, detailing prevalence, disease etiology, as well as current and experimental treatments. IN-DEPTH ANALYSIS OF SOME OF THE MORE DEFINED OR COMMON DISORDERS B-Cell (antibody) Deficiencies T-Cell Deficiencies Combined B- and T-Cell Deficiencies Phagocytic Deficiencies AbstractIntroduction: Primary immune deficiencies are inborn genetic defects in the immune system. It is estimated that at least half a million people worldwide suffer from at least one of these disorders. These patients suffer from recurrent and persistent infections. Gene therapy offers the promise of a potential cure, obliterating the need for antibiotic and immunoglobulin therapy and bone marrow transplants. Scope: * Examines disease etiology, symptoms and treatment options available to patients * Looks at the prevalence and diagnosis of some of the more common and defined conditions and discusses the prognosis for these individuals * Assesses unmet needs in the treatment of primary immune deficiencies and future opportunities, particularly in the area of gene therapy. Report Highlights: Prompt administration of antibiotics at the first signs of infection is vital and those with deficient antibody levels require monthly immunogobulin infusions. Bone marrow transplantation has provided complete remission to some suffering from very severe conditions, such as severe combined immunodeficiency disease (SCID), but global success rates over the past thirty years are only between 6070%. Gene therapy is beginning to show some promise as a viable treatment alternative. The obvious benefit in curing these debilitating illnesses will have to be balanced with the potential risks posed by this new form of treatment. Although entry into the commercial arena is not expected for another few years, the market and growth for gene therapy products will be unprecedented, since application of this technology will have far-reaching implications for a number of other clinical conditions. Reasons to Purchase: * Insightful overview of the key features of primary immune deficiencies, including prevalence, diagnosis and symptoms * Analyze current treatment options and prognosis for some of the more defined and common disorders * Identify unmet needs and learn about future opportunities, especially in gene therapy, which has enormous market potentialGet Full Details About This Report >> |
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