Related by context. Frequent words. (Click for all words.) 72 TTR amyloidosis 71 fatal neurodegenerative 70 neurodegenerative disorder 67 Pulmonary hypertension 65 autosomal dominant 65 progressive degeneration 65 Retinopathy 64 degenerative disorder 64 progressive degenerative 64 Psoriatic arthritis 64 autosomal recessive 64 Leber congenital amaurosis 63 hereditary disorder 63 progressive neurodegenerative disease 63 commercializes innovative biopharmaceuticals 63 Atopic dermatitis 62 hereditary antithrombin deficiency 62 immunodeficiencies 62 Fatty liver 62 spinal muscular atrophy SMA 62 Myelofibrosis 62 neuromuscular dysfunction 62 genetically inherited 62 chronic autoimmune 62 neutropenia thrombocytopenia 61 blood clotting disorder 61 Coeliac disease 61 rheumatoid arthritis lupus 61 retinitis pigmentosa RP 61 demyelinating disease 61 AAT deficiency 61 experimental autoimmune encephalomyelitis 61 Duchenne muscular dystrophy DMD 61 fatal malady 61 ataxia telangiectasia 61 HGPS 61 arthritis gout 61 unknown etiology 61 Pompe Disease 61 inflammation autoimmune diseases 60 muscle degeneration 60 Genital herpes 60 Parkinson disease Alzheimer disease 60 mental retardation cerebral palsy 60 Congenital heart 60 RPE# 60 Lennox Gastaut syndrome 60 Hemorrhagic 60 Major depressive disorder 60 Pervasive Developmental Disorder 60 Muscular dystrophy 60 Friedreich ataxia 60 Multiple sclerosis MS 60 thyroiditis 60 non alcoholic steatohepatitis 60 nephritis 60 Rheumatoid arthritis RA 60 Acromegaly 59 Cytomegalovirus 59 sensory neuropathy 59 chronic degenerative 59 refractory chronic lymphocytic 59 Hemophilia B 59 Diabetic retinopathy 59 Duchenne Muscular Dystrophy DMD 59 Amyotrophic lateral sclerosis 59 Ulcerative colitis 59 lysosomal storage diseases 59 rheumatoid arthritis multiple sclerosis 59 endocrine diseases 59 hyperparathyroidism 59 1 antitrypsin 59 neurobiological disorder 59 glomerulonephritis 59 Metastases 59 degenerative disorders 59 chronic inflammatory bowel 59 active ankylosing spondylitis 59 cell acute lymphoblastic 59 Peripheral neuropathy 59 neurological disorder characterized 59 neurological dysfunction 58 Nausea vomiting 58 Periodontitis 58 acute myelogenous leukemia AML 58 leukemia ALL 58 identify protein biomarkers 58 lung fibrosis 58 metastatic bone 58 progressive neurological disorder 58 endocrine disorder 58 chronic neurological disorder 58 nonsense mutation 58 BioMarin develops 58 enzyme deficiency 58 Mucositis 58 inflammatory autoimmune diseases 58 acquired immunodeficiency syndrome 58 Am J Obstet 58 Cockayne Syndrome 58 Ischemic