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Detailed Listing of Compassionate Allowance Cases

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Detailed Listing of Compassionate Allowance Cases DETAILED LISTING OF COMPASSIONATE ALLOWANCE CASES ACUTE LEUKEMIA Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia Adrenal Carcinoma, Adrenocortical Carcinoma, Adrenocortical ADRENAL CANCER Cancer, Cancer of the Adrenal Cortex, Carcinoma of the Adrenal Cortex Lymphoma - non-Hodgkin's; Lymphocytic lymphoma; ADULT NON-HODGKIN Histiocytic lymphoma; Lymphoblastic lymphoma; Cancer - non- LYMPHOMA Hodgkin's lymphoma ADULT ONSET HUNTINGTON Huntington's chorea; Huntington's Disease DISEASE AGS; Cree Encephalitis; Encephalopathy with Basal Ganglia Calcification; Pseudo-TORCH Syndrome; AICARDI - GOUTIERES Pseudotoxoplasmosis Syndrome; Familial Infantile SYNDROME Encephalopathy with Intracranial Calcification and Chronic Cerebrospinal Fluid Lymphocytosis Alexander Syndrome, Dysmyelogenic Leukodystrophy, Dysmyelogenic Leukodystrophy-Megalobare, Fribrinoid ALEXANDER DISEASE (ALX) - Degeneration of Astrocytes-Infantile type, Fibrinoid NEONATAL AND INFANTILE Leukodystrophy-Infantile type, Hyaline Panneuropathy, Leukodystrophy with Rosenthal Fibers, Megalencephaly with Hyaline Inclusion, Megalencephaly with Hyaline Panneuropathy Allan-Herndon Syndrome; X-linked Intellectual Deficit with ALLAN-HERNDON-DUDLEY Hypotonia; Monocarboxylate Transporter 8 Deficiency; MCT8 SYNDROME Deficiency; MCT8 Specific Thyroid Hormone Cell Transporter Deficiency; MCT8 SLC16A2 Holoprosencephaly; HPE; Holoprosencephaly 1 Alobar; ALOBAR Familial Alobar Holoprosencephaly; Holoprosencephaly HOLOPROSENCEPHALY Sequence Alpers Syndrome; Alpers Progressive Infantile Poliodystrophy; Alpers Huttenlocher Syndrome; AHS; Progressive Neuronal ALPERS DISEASE Degeneration of Childhood; PNDC; Progressive Sclerosing Poliodystrophy; Progressive Infantile Poliodystrophy; Progressive Cerebral Poliodystrophy; Poliodystrophia Cerebri Alpha Mannosidosis Types II/III Early Onset Forms; Alpha-D- mannosidosis; Alpha-mannosidase B deficiency; Alpha- ALPHA MANNOSIDOSIS- mannosidase deficiency; Lysosomal alpha B mannosidosis; TYPES II AND III Lysosomal alpha-D-mannosidase deficiency; Mannosidosis lipid storage disease; Inborn error of metabolism; MAN2B1 Page 1 of 21 DETAILED LISTING OF COMPASSIONATE ALLOWANCE CASES gene abnormality; Mannosidosis ALMS; ALMS1; Alstrom-Halgren Syndrome; Alstrom syndrome ALSTROM SYNDROME 1 ALVEOLAR SOFT PART ASP Sarcoma; ASPS SARCOMA AMEGAKARYOCYTIC Inherited Bone Marrow Failure Syndrome (IBMFS); Congenital THROMBOCYTOPENIA Amegakaryocytic Thrombocytopenia (CAMT) AMYOTROPHIC LATERAL Aran-Duchenne, Gehrig's Disease, Lou Gehrig's Disease, SCLEROSIS (ALS) - ADULT Motor Neuron Disease ANAPLASTIC ADRENAL Adrenal Carcinoma, Anaplastic Adrenal Cancer CANCER - ADULT ANGELMAN SYNDROME AS ANGIOSARCOMA Primary Angiosarcoma; Secondary Angiosarcoma AORTIC ATRESIA Aortic Valve Atresia; Aortic Valve Stenosis Acquired Aplastic Anemia; Anemia Aplastic; Bone Marrow APLASTIC ANEMIA Failure; Idiopathic Aplastic Anemia; Secondary Aplastic Anemia; Severe Aplastic Anemia Astrocytoma Grade III: anaplastic astrocytoma, anaplastic malignant astrocytoma, Astrocytoma Grade IV: glioblastoma ASTROCYTOMA - GRADE III multiforme(GBM), glioblastoma, mixed glioblastoma sarcoma, AND IV gliosarcoma astrocytoma grade IV, giant cell glioblastoma astrocytoma, spongioblastoma multiforme Boder-Sedgwick Syndrome; Louis-Bar Syndrome; Ataxia ATAXIA TELANGIECTASIA Telangiectasia syndrome; A-T; Cerebello-oculocutaneous Telangiectasia ATYPICAL AT/RT; Central Nervous System AT/RT; CNS AT/RT; TERATOID/RHABDOID Malignant AT/RT; Childhood Atypical Teratoid / Rhabdoid TUMOR Tumor; Childhood AT/RT Neuronal Ceroid Lipofuscinoses (NCL); Ceroid Neuronal BATTEN DISEASE Lipofuscinosis (CNL); Spielmeyer-Vogt-Sjogren-Batten disease; Haltia-Santavuori; Jansky-Beilschowsky Beta Thalassemia Major Syndrome; Beta Thalassemia Major BETA THALASSEMIA MAJOR Disease; Thalassemia Major; Cooley Anemia; Cooley Anemia Page 2 of 21 DETAILED LISTING OF COMPASSIONATE ALLOWANCE CASES Disease; Cooley Anemia Syndrome; Erythroblastic Anemia of Childhood; Microcythemia Major; Mediterranean Anemia Major; Beta Zero Thalassemia Infantile Bilateral Optic Atrophy; Bilateral Optic Neuropathy; BILATERAL OPTIC ATROPHY Idiopathic Bilateral Optic Atrophy; Congenital Optic Atrophy; - INFANTILE Pediatric Bilateral Optic Atrophy BILATERAL Malignant Neoplasm, Retina RETINOBLASTOMA Invasive Bladder Cancer, Bladder Carcinoma, Invasive Bladder Carcinoma, Transitional Cell Carcinoma of the Bladder, Transitional Cell Cancer of the Bladder, Squamous Cell BLADDER CANCER Carcinoma of the Bladder, Squamous Cell Cancer of the Bladder, Adenocarcinoma of the Bladder, Urinary Cancer, Urinary Carcinoma Breast Carcinoma (Stage IV), Metastatic Breast Carcinoma, Metastatic Breast Cancer, Ductal Carcinoma of the Breast (Stage IV), Metastatic Ductal Carcinoma, Metastatic Ductal BREAST CANCER Cancer, Lobular Carcinoma of the Breast Stage (IV), Metastatic Lobular Cancer, Metastatic Lobular Carcinoma, Recurrent Breast Cancer Aminoacylase-2 (ACY2) Deficiency, Aspartoacylase (ASPA) Deficiency, Canavan's Leukodystrophy, Spongy Degeneration CANAVAN DISEASE (CD) of the Central Nervous System or Neuroaxis, Van Bogaert- Bertrand Syndrome Cancer of Unknown Primary Origin; CUP; Origin of Unknown CARCINOMA OF UNKNOWN Primary Site; Occult Primary Malignancy; Occult Primary PRIMARY SITE Cancer; Malignant Neoplasms of Unknown site; Malignant Neoplasms of Unknown Origin CARDIAC AMYLOIDOSIS - AL Light Chain Cardiac Amyloidosis; Cardiac Amyloidosis Light TYPE Chain Disease; Primary Cardiac Amyloidosis Caudal Dysplasia Sequence; Caudal Regression Sequence; CAUDAL REGRESSION Caudal Dysgenesis Syndrome; Sacral Regression; Sacral SYNDROME - TYPES III AND Agenesis; Lumbo Sacral Agenesis; Sacral Defect with Anterior IV Meningocele CEREBRO OCULO FACIO Cockayne Syndrome-Classical Type I, Cockayne Syndrome- SKELETAL (COFS) Congenital Type II, Pena Shokeir Syndrome Type II SYNDROME Page 3 of 21 DETAILED LISTING OF COMPASSIONATE ALLOWANCE CASES CEREBROTENDINOUS CTX; Van Bogaert-Scherer-Epstein Disease; Xanthomatosis XANTHOMATOSIS Cerebrotendinous; Cerebral Cholesterosis CHILD LYMPHOBLASTIC LYMPHOMA Non-Hodgkin Lymphoma; Diffuse Large B-cell Lymphoma;B- cell Lymphoma;T-cell Lymphoma; Peripheral T-cell Lymphoma; CHILD LYMPHOMA Follicular Lymphoma; Burkitt Lymphoma; Anaplastic Large Cell Lymphoma Congenital Neuroblastoma; Sympathicoblastoma; Stage IVS CHILD NEUROBLASTOMA Neuroblastoma; Pepper's syndrome; Schwannian Stroma-Poor Neuroblastoma Chondrosarcoma Grade III; Dedifferentiated Chondrosarcoma; CHONDROSARCOMA Mesenchymal Chondrosarcoma CHRONIC IDIOPATHIC Chronic Intestinal Pseudo Obstruction; Intestinal Pseudo INTESTINAL PSEUDO Obstruction; Congenital Idiopathic Intestinal Pseudo OBSTRUCTION Obstruction; Primary Intestinal Pseudo Obstruction CHRONIC MYELOGENOUS Chronic Myeloid Leukemia (Blast phase), CML (Blast phase), LEUKEMIA (CML) - BLAST Chronic Granulocytic Leukemia (Blast phase) PHASE COFFIN-LOWRY SYNDROME Coffin Syndrome; Coffin Lowry Disease Congenital Hereditary Lymphedema; Primary Lymphedema; CONGENITAL LYMPHEDEMA Congenital Primary Lymphedema; Milroy disease CORNELIA DE LANGE Classic Form Cornelia de Lange Syndrome; CDLS1, SYNDROME - CLASSIC Brachmann-De Lange Syndrome; BDLS; CdLS; de Lange FORM Syndrome; Amsterdam Dwarfism; Bushy Syndrome CORTICOBASAL CBD; Cortical-Basal Ganglionic Degeneration; Cortico-Basal DEGENERATION Ganglionic Degeneration; CBGD Jakob-Creutzfeldt Disease, Jakobs Disease, Subacute Spongiform Encephalopathy, Variant (V-CJD) Bovine CREUTZFELDT-JAKOB Spongiform Encephalopathy (BSE), Fatal Familial Insomnia DISEASE (CJD) - ADULT (FFI), Gerstmann-Straussler-Scheinker (GSS) Disease, Prion disease 5p-Syndrome; Cat's Cry Syndrome; Le Jeune Syndrome; CRI DU CHAT SYNDROME Chromosome 5p-Syndrome; 5p deletion syndrome Page 4 of 21 DETAILED LISTING OF COMPASSIONATE ALLOWANCE CASES DE SANCTIS CACCHIONE De Sanctis-Cacchione Syndrome; Xerodermic Idiocy; SYNDROME Xeroderma Pigmentosum with Neurological Manifestation Degos-Kohlmeier Disease; Degos Syndrome; Kohlmeier- DEGOS DISEASE Degos Disease; Malignant Atrophic Papulosis; Kohlmeier disease Severe Myoclonic Epilepsy Of/In Infancy; SMEI Syndrome; DRAVET SYNDROME Epilepsy with Polymorphic Seizures; Polymorphic Epilepsy in Infancy; PMEI EARLY-ONSET ALZHEIMER'S Presenile dementia; Presenile Alzheimer's disease; Young- DISEASE onset Alzheimer's disease; Familial AD; FAD; AD; EOAD Trisomy 18; Chromosome 18; Trisomy 18 Complete; Complete EDWARDS SYNDROME Trisomy 18 Syndrome; Trisomy E Syndrome Eisenmenger Complex; Eisenmenger Disease; Eisenmenger EISENMENGER SYNDROME Reaction; Eisenmenger Physiology Endometrial Stromal Sarcoma Grade III/IV; High Grade ENDOMETRIAL STROMAL Endometrial Stromal Sarcoma; ESS; Undifferentiated Uterine SARCOMA Sarcoma; UUS EMF; Davies Disease; Fibroelastic Endocarditis, Loeffler ENDOMYOCARDIAL Endomyocardial Fibrosis with Eosinophilia; Loeffler Fibroplastic FIBROSIS Parietal Endocarditis EPENDYMOBLASTOMA Childhood Ependymoma; Ependymal Tumors; (CHILD BRAIN CANCER) Neuroectodermal Tumors, Primitive. Erdheim Chester Syndrome; Lipoid Granulomatosis; Non- ERDHEIM CHESTER Langerhans Cell Histiocytosis; Polyostotic Sclerosing DISEASE Histiocytosis Adenocarcinoma of the Esophagus, Squamous cell carcinoma ESOPHAGEAL CANCER of the Esophagus Olfactory Neuroblastoma; Skull Based Olfactory ESTHESIONEUROBLASTOMA Neuroblastoma; Intracranial Olfactory Neuroblastoma; Recurrent Esthesioneuroblastoma Ewing Tumor; Ewing Sarcoma of Bone, Ewing Sarcoma of Soft Tissue; Primitive Neuroectodermal Tumor; PNET; Askin Tumor; EWING
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