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
Adenylosuccinate Lyase Deficiency – Neonatal Form and Type 1 (New)
Adrenal Cancer - with Distant Metastases or Inoperable, Unresectable or Recurrent
Adult Heart Transplant Wait List – Status Levels 1-4
Adult Non-Hodgkin Lymphoma
Adult-Onset Huntington Disease
Aicardi-Goutieres Syndrome
Aicardi Syndrome (New)
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
Au-Kline Syndrome
Bainbridge-Ropers Syndrome
Baraitser-Winter Syndrome (New)
Batten Disease
Beare-Stevenson Cutis Gyrata Syndrome (New)
Beta Thalassemia Major
Bilateral Anophthalmia
Bilateral Optic Atrophy- Infantile
Bilateral Retinoblastoma
Bladder Cancer - with Distant Metastases or Inoperable or Unresectable
Blastic Plasmacytoid Dendritic Cell Neoplasm
Bohring-Opitz Syndrome (New)
Breast Cancer - with Distant Metastases or Inoperable or Unresectable
CACH - Vanishing White Matter Disease – Congenital, Infantile and Early Childhood Onset Forms
Calciphylaxis
Carey-Fineman-Ziter Syndrome
CASK-Related Gene Disorders (New)
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/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
Costello Syndrome
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
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
Harlequin Ichthyosis - Child
Head and Neck Cancers - with Distant Metastasis or Inoperable or Unresectable
Heart Transplant Graft Failure
Hematopoietic Stem Cell Transplantation
Hemophagocytic Lymphohistiocytosis (HLH) - Familial Type
Hepatoblastoma
Hepatocellular Carcinoma
Hepatopulmonary Syndrome
Hepatorenal Syndrome
Hepatosplenic T-Cell Lymphoma (New)
Histiocytic Malignancies
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
Lafora Disease (New)
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 orRecurrent
Lissencephaly
LLMNA-Related Congenital Muscular Dystrophy
Lowe Syndrome
Lymphomatoid Granulomatosis - Grade III
Malignant Brain Stem Gliomas – Childhood
Malignant Ectomesenchymoma
Malignant Gastrointestinal Stromal Tumor
Malignant Germ Cell Tumor
Malignant Migrating Partial Seizures of Infancy (MMPSI) (New)
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
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
OPHN1 Syndrome (New)
Ornithine Transcarbamylase (OTC) Deficiency
Orthochromatic Leukodystrophy with Pigmented Glia
Osteogenesis Imperfecta (OI) - Type II
Osteosarcoma, formerly known as Bone Cancer
Ovarian Cancer – with Distant Metastases or Inoperable or Unresectable
PACS1 Syndrome
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
Pleural Mesothelioma
Pompe Disease – Infantile
Pontocerebellar Hypoplasia
Posterior Cortical Atrophy
Primary Cardiac Sarcoma (New)
Primary Central Nervous System Lymphoma
Primary Effusion Lymphoma
Primary Intracranial Malignant Melanoma (New)
Primary Omental Cancer
Primary Peritoneal Cancer
Primary Progressive Aphasia
Progressive Bulbar Palsy
Progressive Multifocal Leukoencephalopathy
Progressive Muscular Atrophy
Progressive Supranuclear Palsy
Prostate Cancer - Hormone Refractory Disease – or with Visceral Metastases
Pulmonary Amyloidosis - AL Type
Pulmonary Atresia
Pulmonary Kaposi Sarcoma
Rasmussen Encephalitis
Refractory Hodgkin Lymphoma
Renal Amyloidosis - AL Type
Renal Medullary Carcinoma
Renpenning Syndrome
Retinopathy of Prematurity - Stage V, Bilateral
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 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
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
Thymic Carcinoma
Transplant Coronary Artery Vasculopathy
Tricuspid Atresia
Trisomy 9
Turnpenny Fry Syndrome
Ullrich Congenital Muscular Dystrophy
Ureter Cancer - with Distant Metastases or Inoperable, Unresectable or Recurrent
Usher Syndrome - Type I
Uveal Melanoma – with Metastases (New)
Ventricular Assist Device Recipient - Left, Right, or Biventricular
Walker Warburg Syndrome
Warburg Micro Syndrome (New)
WHO Grade III Meningiomas
Wolf-Hirschhorn Syndrome
Wolman Disease
X-Linked Lymphoproliferative Disease
X-Linked Myotubular Myopathy
Xeroderma Pigmentosum
Young-Onset Alzheimer's Disease