Alpha 1 Antitrypsin Deficiency: Key Trends and Statistics Just Published
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NEW YORK--(Business Wire)-- Reportlinker.com announces that a new market research report related to the Healthcare industry is available in its catalogue. Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency To order this report: www.reportlinker.com/p075995/2008/02/Stakeholder-Opinions-Alpha-1- Antitrypsin-Deficiency.html Despite being one of the most common hereditary diseases among Caucasians, low physician awareness and a lack of disease-modifying drugs have led to massive underdiagnosis of the condition. the resulting perceived small number of patients and restricted market is a major obstacle for the development of new treatments. Understand the epidemiology of alpha-1 antitrypsin deficiency and assess the untapped patient potential. Evaluate current strategies for the diagnosis and treatment of this disease.Assess the potential for new drugs and their potential for additional usesGain an understanding of some of the principal challenges in the treatment of alpha 1 antitrypsin deficiency as defined by key opinion leaders. Prevalence estimates have identified 200,000 heterozygous patients in the US and Europe making alpha-1 antitrypsin deficiency one of the most common hereditary disorders in the Western world. However, physician resistance has led to the diagnosis of only a small proportion of the estimated population. Enzyme replacement augmentation therapy is available but lacks randomized, controlled efficacy data. Although widely used in the US, augmentation therapy is unavailable in the UK and Denmark. Patients who receive standard COPD treatment in the UK have comparable mortality rates to US patients who receive augmentation therapy. Inhaled alpha-1 antitrypsin therapy holds promise for the prevention of disease progression but a lack of efficacy data and drug delivery issues are hindering development. Roche has a gamma retinoid agonist, R-667, in Phase II trials and the drug is the only potential therapy that offers lung regeneration through the promotion of alveolar growth. Get an overview of alpha-1 antitrypsin deficient phenotypes and their prevalence in the US and Western European marketsAssess the market opportunity, where established therapies and other developmental compounds fail to address key unmet clinical needsEnhance your commercial positioning through an improved understanding of the alpha-1 antitrypsin deficiency market dynamics ABOUT DATAMONITOR HEALTHCARE 2 About the Respiratory & Infectious Disease (RID) analysis team 2 CHAPTER 1 EXECUTIVE SUMMARY 3 Scope of the analysis 3 Datamonitor insight into the disease market 4 Related reports 4 CHAPTER 2 DISEASE BACKGROUND 6 Alpha-1 antitrypsin deficiency is a hereditary disease found mainly in Caucasians 6 Etiology of alpha-1 antitrypsin deficiency 6 Genetics of Alpha-1 antitrypsin deficiency 8 Epidemiology 10 Emphysema 12 Liver disease 14 Unmet needs 17 Risk factors 17 Smoking 18 Environmental tobacco smoke exposure 19 Occupational exposure 19 Bacterial infections 20 Body mass index 20 CHAPTER 3 DIAGNOSIS 21 Diagnosis of alpha-1 antitrypsin deficiency 21 Lung function 21 Imaging 21 Serum alpha-1 antitrypsin levels 22 Biochemical markers 22 Phenotyping 22 Diagnostic guidelines 22 Genetic screening 23 Increase in age at diagnosis and delay in diagnosis of alpha-1 antitrypsin deficiency 25 Problems with physician knowledge 27 Difficulty in conducting clinical trials in COPD 27 Declining smoking rates 28 CHAPTER 4 TREATMENT OPTIONS 29 Standard COPD therapy 29 Augmentation therapy 29 Therapeutic rationale 29 Market analysis 32 Cost and reimbursement 33 Antibiotic therapy 34 Organ transplant 34 Pulmonary rehabilitation, supplementary oxygen and genetic counseling 35 CHAPTER 5 FUTURE TRENDS 36 Inhaled alpha-1 antitrypsin augmentation therapy 36 Arriva/Hyland 37 Kamada 37 Talecris 37 Recombinant alpha-1 antitrypsin augmentation therapy 37 Gamma retinoid agonists 38 Gene therapy is in the far future 38 Alpha-1 antitrypsin replacement therapy in cystic fibrosis 38 Clinical trial endpoints 39 Continuing medical education 39 The role of patient support groups 40 Neonatal genetic screening 42 Transfer of treatments to the general emphysema population 43 CHAPTER 6 BIBLIOGRAPHY 44 Articles 44 Websites 49 List of Tables Table 1: Alpha-1 antitrypsin levels in common genotypes 9 Table 2: Estimated prevalence of the five main phenotypes of alpha-1 antitrypsin deficiency in selected countries 11 Table 3: Estimated numbers of each of the five main phenotypes of alpha-1 antitrypsin deficiency in selected countries 12 Table 4: Classification of recommendations for genetic testing 24 Table 5: Comparison of augmentation therapies in the US 30 List of Figures Figure 1: Alpha-1 antitrypsin production and activity 8 Figure 2: An example of three-generation pedigree with alpha-1 antitrypsin deficiency 10 Figure 3: Estimated numbers of PiZZ individuals in selected European countries 13 Figure 4: Liver disease in PiZZ patients by age 15 Figure 5: Association between liver dysfunction and age in PiZZ infants 16 Figure 6: Flow diagram of anticipated liver disease outcomes in PiZZ infants 17 Figure 7: Decline in lung function by smoking status 19 Figure 8: Hospital admissions for alpha-1 antitrypsin deficient patients in Norway, 2005 26 Figure 9: SWOT analysis of human, plasma derived augmentation therapy 33 Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency To order this report: www.reportlinker.com/p075995/2008/02/Stakeholder-Opinions-Alpha-1- Antitrypsin-Deficiency.html More market research reports? Go to http://www.reportlinker.com Reportlinker Nicolas, + 33 4 37 65 17 03 email@example.com Copyright Business Wire 2008
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