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Compassionate Allowance

  • Acute Leukemia
  • Adrenal Cancer – with distant metastases or inoperable, unresectable or recurrent
  • Adult Onset Huntington Disease
  • Aicardi Goutieres Syndrome (ALX) – Neonatal and Infantile
  • Alexander Disease (ALX) – Neonatal and Infantile
  • Allan-Herndon-Dudley Syndrome
  • Alobar Holoprosencephaly
  • Alpers Disease
  • Alpha Mannosidosis
  • Alstrom Syndrome
  • Alveolar Soft Part Sarcoma
  • Amegakaryocytic Thrombocytopenia
  • Amyotrophic Lateral Sclerosis (ALS)
  • Anaplastic Adrenal Cancer – with distant metastases or inoperable, unresectable or recurrent
  • Angelman Syndrome
  • Angiosarcoma
  • Aortic Atresia
  • Astrocytoma – Grade III and IV
  • Ataxia Telangiectasia
  • Atypical Teratoid/Rhabdoid Tumor
  • Batten Disease
  • Beta Thalassemia Major
  • Bilateral Optic Atrophy – Infantile
  • Bilateral Retinoblastoma
  • Bladder Cancer – with distant metastases or inoperable or unresectable
  • Bone Cancer – with distant metastases or inoperable or unresectable
  • Breast Cancer – with distant metastases or inoperable or unresectable
  • Canavan Disease (CD)
  • Carcinoma of Unknown Primary Site
  • Caudal Regression Syndrome – Types III and IV
  • Cerebro Oculo Facio Skeletal (COFS) Syndrome
  • Cerebrotendinous Xanthomatosis
  • Child Non Hodgkin Lymphoma
  • Child Neuroblastoma
  • Childhood Severe Combined Immunodeficiency
  • Chondrosarcoma with Multimodal Therapy
  • Chronic Idiopathic Intestinal Pseudo Obstruction
  • Chronic Myelogenous Leukemia (CML) – Blast Phase
  • Coffin-Lowry Syndrome
  • Congenital Lymphedema
  • Cornelia-de-Lange-Syndrome-(Classic-Form)
  • Corticobasal Degeneration
  • Creutzfeldt-Jakob Disease (CJD) – Adult
  • Cri du Chat Syndrome
  • Degos Disease, Systemic
  • De Sanctis Cacchione Syndrome
  • Dravet Syndrome
  • Early-Onset Alzheimer’s Disease
  • Edwards Syndrome (Trisomy 18)
  • Eisenmenger Syndrome
  • Endometrial Stromal Sarcoma
  • Endomyocardial Fibrosis
  • Ependymoblastoma (Child Brain Tumor)
  • Erdheim Chester Disease
  • Esophageal Cancer
  • Esthesioneuroblastoma
  • Ewings Sarcoma
  • Farber’s Disease (FD) – Infantile
  • Fatal Familial Insomnia
  • Fibrodysplasia Ossificans Progressiva
  • Follicular Dendritic Cell Sarcoma with Metastases
  • Friedreichs Ataxia (FRDA)
  • Frontotemporal Dementia (FTD), Picks Disease -Type A – Adult
  • Fryns Syndrome
  • Fucosidosis
  • Fukuyama Congenital Muscular Dystrophy
  • Fulmiant Giant Cell Myocarditis
  • Galactosialidosis
  • Gallbladder Cancer
  • Gaucher Disease (GD) – Type 2
  • Giant Axonal Neuropathy
  • Glioblastoma Multiforme (Adult Brain Tumor)
  • Glioma (Grade III and IV)
  • Glutaric Acidemia Type II (Neonatal)
  • Hallervorden-Spatz Disease
  • Head and Neck Cancers – with distant metastasis or inoperable or uresectable
  • Heart Transplant Graft Failure
  • Heart Transplant Wait List – 1A/1B
  • Hemophagocytic Lymphohistiocytosis (HLH), Familial Type
  • Hepatoblstoma
  • Hepatopulmonary Syndrome
  • Hepatorenal Syndrome
  • Histiocytosis
  • Hoyeaal-Hreidarsson Syndrome
  • Hutchinson Gilford Progeria Syndrome
  • Hydranencephaly
  • Hypophosphatasia Perinatal Lethal Form
  • Hypoplastic Left Heart Syndrome
  • Hypocomplementemic Urticarial Vasculitis
  • I Cell Disease
  • Idiopathic Pulmonary Fibrosis
  • Infantile Bilateral Optic Atrophy
  • Infantile Neuroaxonal Dystrophy (INAD)
  • Infantile Neuronal Ceroid Lipofuscinoses
  • Infantile Free Sialic Acid Storage Disease
  • Inflammatory Breast Cancer (IBC)
  • Intracranial Hemangiopericytoma
  • Jervell and Lange-Nielsen Syndrome
  • Joubert Syndrome
  • Junctional Epidermolysis Bullosa, Lethal Type
  • Juvenile Onset Huntington Disease
  • Kidney Cancer – inoperable or unresectable
  • Krabbe Disease (KD) – Infantile
  • Kufs Disease Type A and B
  • Large Intestine Cancer – with distant metastasis or inoperable, unresectable or recurrent
  • Late Infantile Neuronal Ceroid Lipofuscinoses
  • Left Ventricular Assist Device (LVAD) Recipient
  • Leigh’s Disease
  • Leiomyosarcoma
  • Leptomeningeal Carcinomatosis
  • Lewy Body Dementia
  • Liposarcoma – Metastatic or Recurrent
  • Lissencephaly
  • Liver Cancer
  • Lowe Syndrome
  • Lymphomatoid Granulomatosis Grade III
  • Malignant Ectomesenchymoma
  • Malignant Gastrointestinal Stromal Tumor
  • Malignant Germ Cell Tumor
  • Malignant Melanoma with Metastases
  • Malignant Multiple Sclerosis
  • Mantle Cell Lymphoma (MCL)
  • Malignant Brain Stem Gliomas (Childhood)
  • Malignant Renal Rhabdoid Tumor
  • Maple Syrup Urine Disease
  • Marshall-Smith Syndrome
  • Mastocytosis Type IV
  • Medulloblastoma with Metastasis
  • Classic or Infantile Onset Menkes Disease
  • Merkel Cell Carcinoma with Metastases
  • Merosin Deficient Congenital Muscular Dystrophy
  • Metachromatic Leukodystrophy (MLD) – Late Infantile
  • Mitral Valve Atresia
  • Mixed Dementias
  • MPS I, formally known as Hurler Syndrome
  • MPS II, formally known as Hunter Syndrome
  • MPS III, formally known as Sanfilippo Syndrome
  • Mucosal Malignant Melanoma
  • Multicentric Castleman Disease
  • Multiple System Atrophy
  • Myocolonic Epilepsy and Ragged Red Fibers Syndrome
  • Neonatal Adrenoleukodystrophy
  • Neurodegeneration with Brain Iron Accumulation – Types 1 and 2
  • NFU-1 Mitochondrial Disease
  • Niemann-Pick Disease (NPD) – Type A
  • Niemann-Pick Disease-Type C
  • Nonketotic Hypoglycemia
  • Non-Small Cell Lung Cancer – with metastases to or beyond the hilar nodes or inoperable, unresectable or recurrent
  • Obliterative Bronchiolitis
  • Ohtahara Syndrome
  • Oligodendroglioma Brain Tumor – Grade III
  • Ornithine Transcarbamylase (OTC) Deficiency
  • Orthochromatic Leukodystrophy with Pigmented Glia
  • Osteogenesis Imperfecta (OI) – Type II
  • Ovarian Cancer – with distant metastases or inoperable or unresectable
  • Pallister-Killian Syndrome
  • Pancreatic Cancer
  • Paraneoplastic Pemphigus
  • Parkinsonism Dementia Complex
  • Patau Syndrome (Trisomy 13)
  • Pearson Syndrome
  • Pelizaeus-Merzbacher Disease (Classic Form)
  • Pelizaeus-Merzbacher Disease (Connatal Form)
  • Peritoneal Mesothelioma
  • Peritoneal Mucinous Carcinomatosis
  • Peripheral Nerve Cancer (Metastatic or Recurrent)
  • Perry Syndrome
  • Phelan-McDermid Syndrome
  • Pleural Mesothelioma
  • Pompe Disease – Infantile
  • Primary Cardiac Amyloidosis
  • Primary Central Nervous System Lymphoma
  • Primary Effusion Lymphoma
  • Primary Progressive Aphasia
  • Progressive Bulbar Palsy
  • Progressive Multifocal Leukoencephalopathy
  • Progressive Supranuclear Palsy
  • Pulmonary Atresia
  • Pulmonary Kaposi Sarcoma
  • Rhabdomyosarcoma
  • Rett (RTT) Syndrome
  • Revesz Syndrome
  • Rhizomelic Chondrodysplasia Punctata
  • Roberts Syndrome
  • Salivary Tumors
  • Sandhoff Disease
  • Sanfilippo Syndrome
  • Schindler Disease – Type 1
  • Seckel Syndrome
  • Sjorgen-Larsson Syndrome
  • Single Ventricle
  • Small Cell Cancer (of the Large Intestine, Ovary, Prostate, or Uterus)
  • Small Cell Lung Cancer
  • Small Intestine Cancer – with distant metastases or inoperable, unresectable or recurrent
  • Smith Lemli Opitz Syndrome
  • Soft Tissue Sarcoma – with distant metastases or recurrence
  • Spinal Muscular Atrophy (SMA) – Types 0 And 1
  • Spinocerebellar Ataxia
  • Spinal Nerve Root Cancer
  • Stage V Retinopathy of Prematurity
  • Stiff Person Syndrome
  • Stomach Cancer – with distant metastases or inoperable, unresectable or recurrent
  • Subacute Sclerosis 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, unresectable or recurrent
  • Usher Syndrome
  • Ventricular Assist Device Recipient
  • Walker Warburg Syndrome
  • Wolf Hirschhorn Syndrome
  • Wolman Disease
  • Xeroderma Pigmentosum
  • X-Linked Lymphoproliferative Disease
  • X-Linked Myotubular Myopathy
  • Zellweger Syndrome