Compassionate Allowances Initial List of Conditions
1
Acute Leukemia
2
Adrenal Cancer - with distant metastases or inoperable, unresectable or recurrent
3
Alexander Disease (ALX) - Neonatal and Infantile
4
Alstrom Syndrome
5
Amegakaryocytic Thrombocytopenia
6
Amyotrophic Lateral Sclerosis (ALS)
7
Anaplastic Adrenal Cancer - with distant metastases or inoperable, unresectable or recurrent
8
Astrocytoma - Grade III and IV
9
Ataxia Telangiectasia
10
Batten Disease
11
Bilateral Retinoblastoma
12
Bladder Cancer - with distant metastases or inoperable or unresectable
13
Bone Cancer - with distant metastases or inoperable or unresectable
14
Breast Cancer - with distant metastases or inoperable or unresectable
15
Canavan Disease (CD)
16
Cerebro Oculo Facio Skeletal (COFS) Syndrome
17
Chronic Myelogenous Leukemia (CML) - Blast Phase
18
Creutzfeldt-Jakob Disease (CJD) - Adult
19
Cri du Chat Syndrome
20
Degos Disease, Systemic
21
Early-Onset Alzheimer’s Disease
22
Edwards Syndrome (Trisomy 18)
23
Ependymoblastoma (Child Brain Tumor)
24
Esophageal Cancer
25
Farber's Disease (FD) - Infantile
26
Fibrodysplasia Ossificans Progressiva
27
Friedreichs Ataxia (FRDA)
28
Frontotemporal Dementia (FTD), Picks Disease -Type A - Adult
29
Fukuyama Congenital Muscular Dystrophy
30
Gallbladder Cancer
31
Gaucher Disease (GD) - Type 2
32
Glioblastoma Multiforme (Adult Brain Tumor)
33
Glutaric Acidemia Type II (Neonatal)
34
Head and Neck Cancers - with distant metastasis or inoperable or uresectable
35
Hemophagocytic Lymphohistiocytosis (HLH), Familial Type
36
Idiopathic Pulmonary Fibrosis
37
Infantile Neuroaxonal Dystrophy (INAD)
38
Infantile Neuronal Ceroid Lipofuscinoses
39
Inflammatory Breast Cancer (IBC)
40
Junctional Epidermolysis Bullosa, Lethal Type
41
Kidney Cancer - inoperable or unresectable
42
Krabbe Disease (KD) - Infantile
43
Large Intestine Cancer - with distant metastasis or inoperable, unresectable or recurrent
44
Late Infantile Neuronal Ceroid Lipofuscinoses
45
Leigh’s Disease
46
Lesch-Nyhan Syndrome (LNS)
47
Liver Cancer
48
Mantle Cell Lymphoma (MCL)
49
Maple Syrup Urine Disease
50
Merosin Deficient Congenital Muscular Dystrophy
51
Metachromatic Leukodystrophy (MLD) - Late Infantile
52
Mixed Dementias
53
MPS I, formally known as Hurler Syndrome
54
MPS II, formally known as Hunter Syndrome
55
MPS III, formally known as Sanfilippo Syndrome
56
Mucosal Malignant Melanoma
57
Neonatal Adrenoleukodystrophy
58
Niemann-Pick Disease (NPD) - Type A
59
Niemann-Pick Disease-Type C
60
Non-Small Cell Lung Cancer - with metastases to or beyond the hilar nodes or inoperable, unresectable or recurrent
61
Ornithine Transcarbamylase (OTC) Deficiency
62
Osteogenesis Imperfecta (OI) - Type II
63
Ovarian Cancer - with distant metastases or inoperable or unresectable
64
Pancreatic Cancer
65
Patau Syndrome (Trisomy 13)
66
Peritoneal Mesothelioma
67
Pleural Mesothelioma
68
Pompe Disease - Infantile
69
Primary Progressive Aphasia
70
Progressive Multifocal Leukoencephalopathy
71
Rett (RTT) Syndrome
72
Salivary Tumors
73
Sandhoff Disease
74
Small Cell Cancer (of the Large Intestine, Ovary, Prostate, or Uterus)
75
Small Cell Lung Cancer
76
Small Intestine Cancer - with distant metastases or inoperable, unresectable or recurrent
77
Spinal Muscular Atrophy (SMA) - Types 0 And 1
78
Spinocerebellar Ataxia
79
Stomach Cancer - with distant metastases or inoperable, unresectable or recurrent
80
Subacute Sclerosis Panencephalitis
81
Tay Sachs Disease - Infantile Type
82
Thanatophoric Dysplasia, Type 1
83
Thyroid Cancer
84
Ullrich Congenital Muscular Dystrophy
85
Ureter Cancer - with distant metastases or inoperable, unresectable or recurrent
86
Walker Warburg Syndrome
87
Wolman Disease
88
Zellweger Syndrome
