List of Compassionate Allowance Conditions
Acute Leukemia
Adrenal Cancer - with distant metastases or inoperable, unresectable or recurrent
Adult
Non-Hodgkin Lymphoma
Adult Onset Huntington Disease
Aicardi-Goutieres Syndrome
Alexander Disease
(ALX) - Neonatal and Infantile
Allan-Herndon-Dudley Syndrome
Alobar Holoprosencephaly
Alpers
Disease
Alpha Mannosidosis - Type II and III
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
Aortic Atresia
Aplastic
Anemia
Astrocytoma - Grade III and IV
Ataxia Telangiectasia
Batten Disease
Beta
Thalassemia Major
Bilateral Optic Atrophy- Infantile
Bilateral Retinoblastoma
Bladder 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
Neuroblastoma - with distant metastases or recurrent
Child Non-Hodgkin Lymphoma - recurrent
Child T-Cell
Lymphoblastic Lymphoma
Chondrosarcoma - with multimodal therapy
Chronic Myelogenous Leukemia (CML) -
Blast Phase
Congenital Lymphedema
Cornelia de Lange Syndrome
Corticobasal Degeneration
Creutzfeldt-Jakob
Disease (CJD) – Adult
Cri du Chat Syndrome
Degos Disease - Systemic
DeSanctis 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
Ewing Sarcoma
Farber's
Disease (FD) – Infantile
Fatal Familial Insomnia
Fibrodysplasia Ossificans Progressiva
Follicular
Dendritic Cell Sarcoma - metastatic or recurrent
Friedreichs Ataxia (FRDA)
Frontotemporal Dementia (FTD),
Picks Disease -Type A – Adult
Fryns Syndrome
Fucosidosis - Type 1
Fukuyama Congenital
Muscular Dystrophy
Fulminant Giant Cell Myocarditis
Galactosialidosis - Early and Late Infantile Types
Gallbladder Cancer
Gaucher Disease (GD) - Type 2
Glioblastoma Multiforme (Adult Brain Tumor)
Glioma Grade III and IV
Glutaric Acidemia - Type II
Head and Neck Cancers - with distant
metastasis or inoperable or unresectable
Heart Transplant Graft Failure
Heart Transplant Wait List -
1A/1B
Hemophagocytic Lymphohistiocytosis (HLH) - Familial Type
Hepatoblastoma
Hepatopulmonary
Syndrome
Hepatorenal Syndrome
Histiocytosis Syndromes
Hutchinson-Gilford Progeria Syndrome
Hydranencephaly
Hypocomplementemic Urticarial Vasculitis Syndrome
Hypophosphatasia Perinatal
(Lethal) and Infantile Onset Types
Hypoplastic Left Heart Syndrome
I Cell Disease
Idiopathic
Pulmonary Fibrosis
Infantile Free Sialic Acid Storage Disease
Infantile Neuroaxonal Dystrophy (INAD)
Infantile
Neuronal Ceroid Lipofuscinoses
Inflammatory Breast Cancer (IBC)
Jervell and Lange-Nielsen 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
Lesch-Nyhan
Syndrome (LNS)
Lewy Body Dementia
Lissencephaly
Liver Cancer
Lowe Syndrome
Lymphomatoid
Granulomatosis - Grade III
Malignant Brain Stem Gliomas – Childhood
Malignant Gastrointestinal
Stromal Tumor
Malignant Germ Cell Tumor
Malignant Melanoma - with metastases
Malignant Multiple
Sclerosis
Mantle Cell Lymphoma (MCL)
Maple Syrup Urine Disease
Mastocytosis - Type IV
MECP2
Duplication Syndrome
Medulloblastoma - with metastases
Menkes Disease - Classic or Infantile Onset Form
Merkel Cell Carcinoma - with metastases
Merosin Deficient Congenital Muscular Dystrophy
Metachromatic
Leukodystrophy (MLD) - Late Infantile
Mitral Valve Atresia
Mixed Dementias
MPS I, formerly
known as Hurler Syndrome
MPS II, formerly known as Hunter Syndrome
MPS III, formerly known as Sanfilippo
Syndrome
Mucosal Malignant Melanoma
Multicentric Castleman Disease
Multiple System Atrophy
Myoclonic Epilepsy with Ragged Red Fibers Syndrome
Neonatal Adrenoleukodystrophy
Nephrogenic
Systemic Fibrosis
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 Hyperglycinemia
Non-Small Cell Lung Cancer - with metastases to or beyond the hilar nodes or inoperable, unresectable or recurrent
Obliterative
Bronchiolitis
Ohtahara Syndrome
Ornithine Transcarbamylase (OTC) Deficiency
Orthochromatic
Leukodystrophy with Pigmented Glia
Osteogenesis Imperfecta (OI) - Type II
Osteosarcoma, formerly known
as Bone Cancer - with distant metastases or inoperable or unresectable
Ovarian Cancer – with distant
metastases or inoperable or unresectable
Pancreatic Cancer
Paraneoplastic Pemphigus
Patau
Syndrome (Trisomy 13)
Pearson Syndrome
Pelizaeus-Merzbacher Disease-Classic Form
Pelizaeus-Merzbacher
Disease-Connatal Form
Peripheral Nerve Cancer - metastatic or recurrent
Peritoneal Mesothelioma
Peritoneal
Mucinous Carcinomatosis
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 Multifocal Leukoencephalopathy
Progressive
Supranuclear Palsy
Pulmonary Atresia
Pulmonary Kaposi Sarcoma
Retinopathy of Prematurity
- Stage V
Rett (RTT) Syndrome
Rhabdomyosarcoma
Rhizomelic Chondrodysplasia Punctata
Roberts
Syndrome
Salivary Tumors
Sandhoff Disease
Schindler Disease - Type 1
Severe Combined
Immunodeficiency - Childhood
Single Ventricle
Sinonasal Cancer
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
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
The ALS/Parkinsonism Dementia Complex
Thyroid Cancer
Transplant Coronary
Artery Vasculopathy
Tricuspid Atresia
Ullrich Congenital Muscular Dystrophy
Ureter Cancer
- with distant metastases or inoperable, unresectable or recurrent
Usher Syndrome - Type I
Walker Warburg
Syndrome
Wolf-Hirschhorn Syndrome
Wolman Disease
Xeroderma Pigmentosum
Zellweger
Syndrome