A new research document with title ‘Fabry Disease – Pipeline Analysis 2022’ covering detailed analysis, Competitive landscape, forecast and strategies. The study covers geographic analysis that includes regions and important players/vendors such as Amicus Therapeutics (Fabry Gene Therapy); Freeline (FTL190); Greenovation Biotech (Moss-aGal); Idorsia Pharmaceuticals (Lucerastat); Moderna (mRNA-3630); Pharming (PGN005); Protalix Biotherapeutics (Pegunigalsidase alfa); Resverlogix Corp (Apabetalone); Sangamo Therapeutics (ST-920) and Sanofi Genzyme (Venglustat). The report will help user gain market insights, future trends and growth prospects for forecast period of 2022-2028
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Fabry Disease (also known as Anderson Fabry Disease) is a progressive X linked inherited genetic disorder of glycosphingolipid metabolism due to deficient or absent lysosomal α-galactosidase A activity. It is a devastating inborn error of metabolism with, particularly in the early stages, being played by cellular dysfunction and microvascular pathology being induced by lysosomal glycosphingolipid deposition.
Recombinant alpha-galactosidase A (alpha-Gal A), the enzyme that is deficient in patients with Fabry disease, and migalastat hydrochloride, an oral pharmacological chaperone that facilitates trafficking of alpha-Gal A to lysosomes, are therapeutic options for eligible individuals. The therapeutic management primarily focuses on the control of blood pressure, lipids, and proteinuria. ACE inhibitors and/or blockers are used in patients with proteinuria. Hypertension and hypercholesterolemia are be managed appropriately. The prophylaxis with anticoagulants is important in patients who have had ischemic attacks or stroke, and permanent cardiac pacing are considered in high-risk patients.
Emerging treatment strategies for Fabry disease involve the development of small molecule compounds, which are widely used for the treatment of a variety of diseases. Such small molecules account for approximately 80-90% of marketed drugs. Although enzyme replacement therapy is effective, there is a need for other therapeutic strategies, which can either serve as primary or supplemental treatments. Small molecule drug discovery is promising, as it could lead to new treatments for Fabry disease. The discovery of non-inhibitory chaperones, activators, or inhibitors of the enzymes that degrade glycosphingolipids, would be a breakthrough.
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1. REPORT INTRODUCTION
1.1 Objective of the Study 8
1.2 Secondary Research 8
1.3 Scope of the report: 8
2. DISEASE OVERVIEW 9
2.1 Introduction 9
2.2 Etiology 10
2.3 Classification 11
2.4 Signs and Symptoms 12
2.5 Pathogenesis 14
2.5.1 Metabolic Defect 14
2.5.2 The Molecular Basis 15
2.5.3 Genotype/Phenotype Correlation 18
2.6 Diagnosis 19
2.6.1 Biochemical Diagnosis: 19
2.6.1.1 Measurement of a-Gal Activity in Cultivated Fibroblasts, Plasma, or Leukocytes 19
2.6.1.2 Measurement of α-Gal A Activity in Blood Spot Filter Paper 20
2.6.1.3 GL-3 Levels 20
2.6.2 Molecular Diagnosis 21
2.6.3 Molecular Analysis 21
2.7 Treatment 23
2.7.1 Clinical management of pediatric patients 24
2.7.1 Management of Fabry related pain in paediatric patients 24
2.7.1.1 Trigger management 24
2.7.1.2 Symptomatic therapy 25
2.7.1.2.1 Management of neuropathic pain 25
2.7.1.2.2 Management of psychological disturbances 26
2.7.2 Treatment Algorithm for the management of neuropathic pain in paediatric Fabry Disease patients 27
2.7.2.1 Enzyme replacement therapy 28
2.7.2.2 Recommendations for ERT initiation 29
2.7.3 Management in adults 30
2.7.3.1 Available Enzyme Replacement therapies 31
2.7.3.2 Ini
….Continued
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