List of Compassionate Allowance Medical Conditions

(CAL Diseases, Disorders, and Impairments)

1p36 Deletion Syndrome

Acute Leukemia

Adrenal Cancer - with distant metastases or inoperable, unresectable or recurrent

Adult Heart Transplant Wait List – Status Levels 1-4 (New)

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

ALS/Parkinsonism Dementia Complex

Alstrom Syndrome

Alveolar Soft Part Sarcoma

Amegakaryocytic Thrombocytopenia

Amyotrophic Lateral Sclerosis (ALS)

Anaplastic Adrenal Cancer - Adult with distant metastases or inoperable, unresectable or recurrent

Anaplastic Ependymoma

Angelman Syndrome

Angioimmunoblastic T-Cell Lymphoma

Angiosarcoma

Aortic Atresia

Aplastic Anemia

Astrocytoma - Grade III and IV

Ataxia Telangiectasia

Atypical Teratoid/Rhabdoid Tumor

Bainbridge-Ropers Syndrome (New)

Batten Disease

Beta Thalassemia Major

Bilateral Optic Atrophy- Infantile

Bilateral Retinoblastoma

Bladder Cancer - with distant metastases or inoperable or unresectable

Blastic Plasmacytoid Dendritic Cell Neoplasm

Breast Cancer - with distant metastases or inoperable or unresectable

CACH--Vanishing White Matter Disease-Infantile and Childhood Onset Forms

Calciphylaxis

Canavan Disease (CD)

Carcinoma of Unknown Primary Site

Cardiac Amyloidosis- AL Type

Caudal Regression Syndrome - Types III and IV

CDKL5 Deficiency Disorder

Cerebro Oculo Facio Skeletal (COFS) Syndrome

Cerebrotendinous Xanthomatosis

Costello Syndrome (New)

Charlevoix-Saguenay Spastic Ataxia

Child Heart Transplant Wait List – Status Levels 1A/1B

Child Lymphoblastic Lymphoma

Child Lymphoma

Child Neuroblastoma - with distant metastases or recurrent

Cholangiocarcinoma

Chondrosarcoma - with multimodal therapy

Choroid Plexus Carcinoma

Chronic Idiopathic Intestinal Pseudo Obstruction

Chronic Myelogenous Leukemia (CML) - Blast Phase

CIC-rearranged Sarcoma

Coffin-Lowry Syndrome

Congenital Lymphedema

Congenital Myotonic Dystrophy

Congenital Zika Syndrome

Cornelia de Lange Syndrome - Classic Form

Corticobasal Degeneration

Creutzfeldt-Jakob Disease (CJD) – Adult

Cri du Chat Syndrome

Degos Disease - Systemic

DeSanctis Cacchione Syndrome

Desmoplastic Mesothelioma

Desmoplastic Small Round Cell Tumors

Dravet Syndrome

Duchenne Muscular Dystrophy- Adult

Early-Onset Alzheimer’s Disease

Edwards Syndrome (Trisomy 18)

Eisenmenger Syndrome

Endometrial Stromal Sarcoma

Endomyocardial Fibrosis

Ependymoblastoma (Child Brain Cancer)

Erdheim Chester Disease

Esophageal Cancer

Esthesioneuroblastoma

Ewing Sarcoma

Farber's Disease (FD) – Infantile

Fatal Familial Insomnia

Fibrodysplasia Ossificans Progressiva

Fibrolamellar Cancer

Follicular Dendritic Cell Sarcoma - metastatic or recurrent

FOXG1 Syndrome

Friedreich's Ataxia (FRDA)

Frontotemporal Dementia (FTD), Pick's 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

Gerstmann-Straussler-Scheinker Disease

Giant Axonal Neuropathy

Glioblastoma Multiforme (Brain Cancer)

Glioma - Grade III and IV

Glutaric Acidemia - Type II

GM1 Gangliosidosis - Infantile and Juvenile Forms

Head and Neck Cancers - with distant metastasis or inoperable or unresectable

Heart Transplant Graft Failure

Hemophagocytic Lymphohistiocytosis (HLH) - Familial Type

Hepatoblastoma

Hepatocellular Carcinoma

Hepatopulmonary Syndrome

Hepatorenal Syndrome

Histiocytic Malignancies (New)

Histiocytosis Syndromes

Hoyeraal-Hreidarsson Syndrome

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)

Intracranial Hemangiopericytoma

Jervell and Lange-Nielsen Syndrome

Joubert Syndrome

Junctional Epidermolysis Bullosa - Lethal Type

Juvenile Onset Huntington Disease

Kidney Cancer - inoperable or unresectable

Kleefstra Syndrome

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

Leber Congenital Amaurosis

Leigh’s Disease

Leiomyosarcoma

Leptomeningeal Carcinomatosis

Lesch-Nyhan Syndrome (LNS)

Lewy Body Dementia

Liposarcoma - metastatic or recurrent

Lissencephaly

Lowe Syndrome

Lymphomatoid Granulomatosis - Grade III

Malignant Brain Stem Gliomas – Childhood

Malignant Ectomesenchymoma

Malignant Gastrointestinal Stromal Tumor

Malignant Germ Cell Tumor

Malignant Multiple Sclerosis

Malignant Renal Rhabdoid Tumor

Mantle Cell Lymphoma (MCL)

Maple Syrup Urine Disease

Marshall-Smith Syndrome

Mastocytosis - Type IV

MECP2 Duplication Syndrome

Medulloblastoma

Megacystis Microcolon Intestinal Hypoperistalsis Syndrome

Megalencephaly Capillary Malformation Syndrome

Menkes Disease - Classic or Infantile Onset Form

Merkel Cell Carcinoma - with metastases

Merosin Deficient Congenital Muscular Dystrophy

Metachromatic Leukodystrophy (MLD) - Late Infantile

Metastatic Endometrial Adenocarcinoma

Microvillus Inclusion Disease – Child

Mitral Valve Atresia

Mixed Dementias

Mowat-Wilson Syndrome

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

Myelodysplastic Syndrome with Excess Blasts

Myoclonic Epilepsy with Ragged Red Fibers Syndrome

Neonatal Adrenoleukodystrophy

Neonatal Marfan Syndrome (New)

Nephrogenic Systemic Fibrosis

Neurodegeneration with Brain Iron Accumulation - Types 1 and 2

NFU-1 Mitochondrial Disease

Nicolaides-Baraister Syndrome

Niemann-Pick Disease (NPD) - Type A

Niemann-Pick Disease-Type C

Nonketotic Hyperglycinemia

Non-Small Cell Lung Cancer

NUT Carcinoma

Obliterative Bronchiolitis

Ohtahara Syndrome

Oligodendroglioma Brain Cancer- Grade III

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

PACS1 Syndrome (New)

Pallister-Killian Syndrome

Pancreatic Cancer

Paraneoplastic Cerebellar Degeneration

Paraneoplastic Pemphigus

Patau Syndrome (Trisomy 13)

Pearson Syndrome

Pelizaeus-Merzbacher Disease-Classic Form

Pelizaeus-Merzbacher Disease-Connatal Form

Pericardial Mesothelioma

Peripheral Nerve Cancer - metastatic or recurrent

Peritoneal Mesothelioma

Peritoneal Mucinous Carcinomatosis

Perry Syndrome

Pfeiffer Syndrome - Types II and III

Phelan-McDermid Syndrome

Pineoblastoma - Childhood

Pitt Hopkins Syndrome

Plasmablastic Lymphoma (New)

Pleural Mesothelioma

Pompe Disease – Infantile

Pontocerebellar Hypoplasia

Posterior Cortical Atrophy

Primary Central Nervous System Lymphoma

Primary Effusion Lymphoma

Primary Omental Cancer

Primary Peritoneal Cancer

Primary Progressive Aphasia

Progressive Bulbar Palsy

Progressive Multifocal Leukoencephalopathy

Progressive Supranuclear Palsy

Prostate Cancer - Hormone Refractory Disease – or with visceral metastases

Pulmonary Atresia

Pulmonary Kaposi Sarcoma

Refractory Hodgkin Lymphoma

Renal Amyloidosis - AL Type

Renal Medullary Carcinoma (New)

Renpenning Syndrome

Retinopathy of Prematurity - Stage V

Rett (RTT) Syndrome

Revesz Syndrome

Rhabdomyosarcoma

Rhizomelic Chondrodysplasia Punctata

Richter Syndrome

Roberts Syndrome

Rubinstein-Taybi Syndrome

Salivary Cancers

Sandhoff Disease

Sarcomatoid Carcinoma of the Lung - Stages II - IV

Sarcomatoid Mesothelioma

Schindler Disease - Type 1

SCN8A Related Epilepsy with Encephalopathy

Seckel Syndrome

Secondary Adenocarcinoma of the Brain

Severe Combined Immunodeficiency - Childhood

Single Ventricle

Sinonasal Cancer

Sjogren-Larsson Syndrome

Skin Malignant Melanoma with Metastases

Small Cell Cancer - Large Intestine

Small Cell Cancer - Prostate

Small Cell Cancer - Thymus

Small Cell Cancer of the Female Genital Tract

Small Cell Lung Cancer

Small Intestine Cancer - with distant metastases or inoperable, unresectable or recurrent

Smith Lemli Opitz Syndrome

Snijders Blok-Campeau Syndrome (New)

Soft Tissue Sarcoma - with distant metastases or recurrent

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

Superficial Siderosis of the Central Nervous System

SYNGAP1-related NSID

Tabes Dorsalis

Tay Sachs Disease - Infantile Type

Taybi-Linder Syndrome

Tetrasomy 18p

Thanatophoric Dysplasia - Type 1

Thyroid Cancer

Transplant Coronary Artery Vasculopathy

Tricuspid Atresia

Trisomy 9

Ullrich Congenital Muscular Dystrophy

Ureter Cancer - with distant metastases or inoperable, unresectable or recurrent

Usher Syndrome - Type I

Ventricular Assist Device Recipient - Left, Right, or Biventricular

Walker Warburg Syndrome

Wolf-Hirschhorn Syndrome

Wolman Disease

X-Linked Lymphoproliferative Disease

X-Linked Myotubular Myopathy

Xeroderma Pigmentosum

Zellweger Syndrome