| 1 |
Acute Leukemia |
| 2 |
Adrenal Cancer - with distant metastases or inoperable, unresectable or recurrent |
| 3 |
Alexander Disease (ALX) - Neonatal and Infantile |
| 4 |
Amyotrophic Lateral Sclerosis (ALS) |
| 5 |
Anaplastic Adrenal Cancer - with distant metastases or inoperable, unresectable or recurrent |
| 6 |
Astrocytoma - Grade III and IV |
| 7 |
Bladder Cancer - with distant metastases or inoperable or unresectable |
| 8 |
Bone Cancer - with distant metastases or inoperable or unresectable |
| 9 |
Breast Cancer - with distant metastases or inoperable or unresectable |
| 10 |
Canavan Disease (CD) |
| 11 |
Cerebro Oculo Facio Skeletal (COFS) Syndrome |
| 12 |
Chronic Myelogenous Leukemia (CML) - Blast Phase |
| 13 |
Creutzfeldt-Jakob Disease (CJD) - Adult |
| 14 |
Ependymoblastoma (Child Brain Tumor) |
| 15 |
Esophageal Cancer |
| 16 |
Farber's Disease (FD) - Infantile |
| 17 |
Friedreichs Ataxia (FRDA) |
| 18 |
Frontotemporal Dementia (FTD), Picks Disease -Type A - Adult |
| 19 |
Gallbladder Cancer |
| 20 |
Gaucher Disease (GD) - Type 2 |
| 21 |
Glioblastoma Multiforme (Brain Tumor) |
| 22 |
Head and Neck Cancers - with distant metastasis or inoperable or uresectable |
| 23 |
Infantile Neuroaxonal Dystrophy (INAD) |
| 24 |
Inflammatory Breast Cancer (IBC) |
| 25 |
Kidney Cancer - inoperable or unresectable |
| 26 |
Krabbe Disease (KD) - Infantile |
| 27 |
Large Intestine Cancer - with distant metastasis or inoperable, unresectable or recurrent |
| 28 |
Lesch-Nyhan Syndrome (LNS) |
| 29 |
Liver Cancer |
| 30 |
Mantle Cell Lymphoma (MCL) |
| 31 |
Metachromatic Leukodystrophy (MLD) - Late Infantile |
| 32 |
Niemann-Pick Disease (NPD) - Type A |
| 33 |
Non-Small Cell Lung Cancer - with metastases to or beyond the hilar nodes or inoperable, unresectable or recurrent |
| 34 |
Ornithine Transcarbamylase (OTC) Deficiency |
| 35 |
Osteogenesis Imperfecta (OI) - Type II |
| 36 |
Ovarian Cancer - with distant metastases or inoperable or unresectable |
| 37 |
Pancreatic Cancer |
| 38 |
Peritoneal Mesothelioma |
| 39 |
Pleural Mesothelioma |
| 40 |
Pompe Disease - Infantile |
| 41 |
Rett (RTT) Syndrome |
| 42 |
Salivary Tumors |
| 43 |
Sandhoff Disease |
| 44 |
Small Cell Cancer (of the Large Intestine, Ovary, Prostate, or Uterus) |
| 45 |
Small Cell Lung Cancer |
| 46 |
Small Intestine Cancer - with distant metastases or inoperable, unresectable or recurrent |
| 47 |
Spinal Muscular Atrophy (SMA) - Types 0 And 1 |
| 48 |
Stomach Cancer - with distant metastases or inoperable, unresectable or recurrent |
| 49 |
Thyroid Cancer |
| 50 |
Ureter Cancer - with distant metastases or inoperable, unresectable or recurrent |