Related by context. Frequent words. (Click for all words.) 65 degenerative disorder 63 muscle degeneration 63 progressive degenerative 63 fatal neurodegenerative 63 Leber congenital amaurosis 62 neurodegenerative disorder 61 genetically inherited 60 progressive degeneration 60 Retinoblastoma 60 progressive neurological disorder 60 Muscular dystrophy 60 Retinopathy 60 Angelman syndrome 59 nephritis 59 Fanconi anemia 59 epidermolysis bullosa 59 myotonic dystrophy 59 incurable lung 59 Angelman Syndrome 59 Anorexia nervosa 59 Cystic fibrosis 58 DiGeorge syndrome 58 genetic disorder 58 enzyme deficiency 58 congenital disorders 58 prosopagnosia 58 NF2 57 Hyperthyroidism 57 Prader Willi syndrome 57 Macular degeneration 57 Ulcerative colitis 57 progressive neurodegenerative disease 57 rhabdomyosarcoma 57 Testicular cancer 57 genetic underpinnings 57 metabolic disorder 57 bone deformities 57 Duchenne muscular dystrophy DMD 56 myelodysplasia 56 Duchenne Muscular Dystrophy DMD 56 developmental abnormalities 56 infantile spasms 56 acute promyelocytic leukemia 56 blood clotting disorder 56 autosomal dominant 56 neurodevelopmental disorder 56 genetic abnormality 56 motor neuron disease 56 Polycystic Kidney Disease 56 biliary atresia 56 retinal degeneration 56 Alzheimers disease 56 ependymoma 56 FTLD 55 Genital herpes 55 Fragile X Syndrome 55 congenital disorder 55 chromosomal disorder 55 dystrophy 55 Krabbe disease 55 genetic defect 55 osteogenesis imperfecta 55 spinal muscular atrophy 55 Wilms tumor 55 Friedreich ataxia 55 hereditary disorder 55 cystic fibrosis 55 neurodegenerative disease 55 Cystinosis 55 recurrent infections 55 thyroid dysfunction 55 AAT deficiency 55 Rett syndrome 55 rheumatic disease 55 muscular dystrophies 55 Multiple sclerosis MS 55 Congenital heart 54 Urinary tract infections 54 Malignant melanoma 54 Esophageal cancer 54 Progeria 54 Autoimmune diseases 54 Irritable bowel syndrome 54 degenerative disease 54 Glioblastoma 54 mitochondrial disease 54 hyperparathyroidism 54 Gliomas 54 Diabetic retinopathy 54 Hip fractures 54 wet macular degeneration 54 Tay Sachs disease 54 adrenal cancer 54 diagnosed 54 Spina bifida 54 genetic mutation 54 brain lesions 54 commercializes innovative biopharmaceuticals 54 disease 54 retinitis pigmentosa 54 Emphysema