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Compassionate Allowances Conditions 1-225 Batten Disease Degos Disease Glioblastoma Multiforme (Brain Acute Leukemia Cancer) Beta Thalassemia Major Dravet Syndrome Adrenal Cancer with distant Glioma Grade III and IV metasteses or inoperable, unresectable, or Bilateral Optic Atrophy - Infantile Early-Onset Alzheimer’s Disease recurrent Glutaric Acidemia - Type II Bilateral Retinoblastoma Edwards Syndrome Adult Non-Hodgkin Lymphoma Head and Neck Cancers Bladder Cancer with distant Eisenmenger Syndrome Adult Onset Huntington Disease metastases or inoperable or unresectable Heart Transplant Graft Failure Endometrial Stromal Sarcoma Aicardi-Goutieres Syndrome Breast Cancer with distant metastases Heart Transplant Wait List - or inoperable or unresectable Endomyocardial Fibrosis Alexander Disease (ALX) - 1A/1B Neonatal and Infantile Ependymoblastoma (Child Brain Canavan Disease (CD) Hemophagocytic Cancer) Allan-Herndon-Dudley Carcinoma of Unknown Primary Lymphohistiocytosis - Familial Syndrome Site Erdheim Chester Disease Type Alobar Holoprosencephaly Cardiac Amyloidosis – AL Type Esophageal Cancer Hepatoblastoma Alpers Disease Caudal Regression Syndrome - Esthesioneuroblastoma Hepatopulmonary Syndrome Types III and IV Alpha Mannosidosis - Types II and Ewing Sarcoma Hepatorenal Syndrome III Cerebro Oculo Facio Skeletal Farber Disease (FD) - Infantile Histiocytosis Syndromes ALS/Parkinsonism Dementia (COFS) Syndrome Complex Cerebrotendinous Xanthomatosis Fatal Familial Insomnia Hoyeaal-Hreidarsson Syndrome Alstrom Syndrome Child Lymphoblastic Lymphoma Fibrodysplasia Ossificans Hutchinson-Gilford Progeria Progressiva Syndrome Alveolar Soft Part Sarcoma Child Lymphoma Follicular Dendritic Cell Hydranencephaly Amegakaryocytic Child Neuroblastoma with distant Sarcoma metastases or recurrent Hypocomplementemic Urticarial Thrombocytopenia Friedreichs Ataxia (FRDA) Vasculitis Syndrome Chondrosarcoma Amyotrophic Lateral Sclerosis Frontotemporal Dementia (FTD), Hypophosphatasia - Perinatal (ALS) - Adult Chronic Idiopathic Intestinal Picks Disease -Type A - Adult (Lethal) and Infantile Onset Pseudo Obstruction Types Anaplastic Adrenal Cancer – Fryns Syndrome Adult with distant metasteses or Chronic Myelogenous Leukemia Hypoplastic Left Heart inoperable, unresectable or recurrent (CML) - Blast Phase Fucosidosis - Type I Syndrome Angelman Syndrome Fukuyama Congenital Muscular Coffin-Lowry Syndrome I Cell disease Dystrophy Angiosarcoma Congenital Lymphedema Idiopathic Pulmonary Fibrosis Fulminant Giant Cell Aortic Atresia Cornelia de Lange Syndrome - Myocarditis Infantile Free Sialic Acid Storage Classic Form Aplastic Anemia Disease Galactosialidosis - Early and Late Corticobasal Degeneration Astrocytoma - Grade III and IV Infantile Types Infantile Neuroaxonal Dystrophy Creutzfeldt-Jakob Disease (CJD) Gallbladder Cancer (INAD) Ataxia Telangiectasia - Adult Gaucher Disease (GD) - Type 2 Infantile Neuronal Ceroid Atypical Teratoid/Rhabdoid Cri du Chat Syndrome Lipofuscinoses Tumor Giant Axonal Neuropathy De Sanctis Cacchione Syndrome Inflammatory Breast Cancer Updated April 6, 2017 Compassionate Allowances Conditions 1-225 Intracranial Malignant Renal Rhabdoid Niemann-Pick Disease-Type C Pompe Disease - Infantile Hemangiopericytoma Tumor Nonketotic Hyperglycinemia Primary Central Nervous System Jervell and Lange-Nielsen Mantle Cell Lymphoma (MCL) Lymphoma Non-Small Cell Lung Cancer Syndrome Maple Syrup Urine Disease Primary Effusion Lymphoma Obliterative Bronchiolitis Joubert Syndrome Marshall-Smith Syndrome Primary Progressive Aphasia Ohtahara Syndrome Junctional Epidermolysis Bullosa Mastocytosis - Type IV Progressive Bulbar Palsy - Lethal Type Oligodendroglioma Brain Cancer MECP2 Duplication Syndrome - Grade III Progressive Multifocal Juvenile Onset Huntington Leukoencephalopathy Disease Medulloblastoma - with metastases Ornithine Transcarbamylase (OTC) Deficiency Progressive Supranuclear Palsy Kidney Cancer Menkes Disease - Classic or Infantile Onset Form Orthochromatic Leukodystrophy Prostate Cancer - Hormone Krabbe Disease (KD) - Infantile with Pigmented Glia Refractory Disease - or with visceral Merkel Cell Carcinoma - with Kufs Disease Type A and B metastases metastases Osteogenesis Imperfecta (OI) - Pulmonary Atresia Large Intestine Cancer Merosin Deficient Congenital Type II Late Infantile Neuronal Ceroid Muscular Dystrophy Osteosarcoma, formerly known as Pulmonary Kaposi Sarcoma Lipofuscinoses Bone Cancer - with distant metastases, or Metachromatic Leukodystrophy inoperable or unresectable Retinopathy of Prematurity - Stage Leigh’s Disease (MLD) - Late Infantile V Ovarian Cancer - with distant Leiomyosarcoma Mitral Valve Atresia metastases or inoperable or unresectable Rett (RTT) Syndrome Leptomeningeal Carcinomatosis Mixed Dementia Pallister-Killian Syndrome Revesez Syndrome Lesch-Nyhan Syndrome (LNS) MPS I Hurler Syndrome Pancreatic Cancer Rhabdomyosarcoma Lewy Body Dementia MPS II Hunter Syndrome Paraneoplastic Pemphigus Rhizomelic Chondrodysplasia Punctata Liposarcoma - metastatic or recurrent MPS III Sanfilippo Syndrome Patau Syndrome Roberts Syndrome LissencephalyLiver Cancer Mucosal Malignant Melanoma Pearson Syndrome Salivary Cancers Liver Cancer Multicentric Castleman Disease Pelizaeus-Merzbacher Disease - Classic Form Sandhoff Disease Lowe Syndrome Multiple System Atrophy Pelizaeus-Merzbacher Disease - Schindler Disease - Type 1 Lymphomatoid Granulomatosis - Myoclonic Epilepsy with Ragged Connatal Form Grade III Red Fibers Syndrome Seckel Syndrome Peripheral Nerve Cancer – Neonatal Adrenoleukodystrophy metastatic or recurrent Severe Combined Malignant Brain Stem Gliomas - Immunodeficiency - Childhood Childhood Nephrogenic Systemic Fibrosis Peritoneal Mesothelioma Single Ventricle Malignant Ectomesenchymoma Neurodegeneration with Brain Peritoneal Mucinous Iron Accumulation - Type 1 and Carcinomatosis Sinonasal Cancer Malignant Gastrointestinal Type 2 Stromal Tumor Perry Syndrome Sjögren-Larsson Syndrome NFU-1 Mitochondrial Disease Malignant Germ Cell Tumor Phelan- McDermid Syndrome Skin Malignant Melanoma Niemann-Pick Disease (NPD) - Small Cell Cancer (Large Malignant Multiple Sclerosis Type A Pleural Mesothelioma Intestine, Prostate, or Thymus) Updated April 6, 2017 Compassionate Allowances Conditions 1-225 Small Cell Cancer of the Female Wolf-Hirschhorn Syndrome Genital Tract Wolman Disease Small Cell Lung Cancer X-Linked Lymphoproliferative Small Intestine Cancer Disease Smith Lemli Opitz Syndrome X-Linked Myotubular Myopathy Soft Tissue Sarcoma - with distant Xeroderma Pigmentosum metastases or recurrent Zellweger Syndrome Spinal Muscular Atrophy (SMA) - Types 0 And 1 Spinal Nerve Root Cancer - metastatic or recurrent Spinocerebellar Ataxia Stiff Person Syndrome Stomach Cancer - with distant metastases or inoperable, unresectable or recurrent Subacute Sclerosing Panencephalitis Tabes Dorsalis Tay Sachs Disease - Infantile Type Thanatophoric Dysplasia - Type 1 Thyroid Cancer Transplant Coronary Artery Vasculopathy Tricuspid Atresia Ullrich Congenital Muscular Dystrophy Ureter Cancer – with distant metastases or inoperable or unresectable or recurrent Usher Syndrome - Type I Ventricular Assist Device Recipient – Left, Right, or Biventricular Walker Warburg Syndrome Updated April 6, 2017 .
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