Compassionate Allowances Conditions
CAL Conditions
1 A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
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)
Beta Thalassemia MajorBilateral 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
Charlevoix-Saguenay Spastic Ataxia
Child Heart Transplant Wait List – Status Levels 1A/1BChild Lymphoma
Child Neuroblastoma - with distant metastases or recurrent
Cholangiocarcinoma
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
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 InsomniaFibrodysplasia 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 CancerGaucher 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 FailureHemophagocytic 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
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 SyndromeKrabbe Disease (KD) – Infantile
Kufs Disease - Type A and B
Large Intestine Cancer - with distant metastasis or inoperable, unresectable or recurrent
Late Infantile Neuronal Ceroid LipofuscinosesLeber 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
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
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
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
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
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
Sjogren-Larsson Syndrome
Skin Malignant Melanoma with Metastases
Small Cell Cancer (Large Intestine, Prostate or 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
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
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 recurrentUsher Syndrome - Type I
Ventricular Assist Device Recipient - Left, Right, or Biventricular
Wolman Disease