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