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Compassionate Allowance Conditions
Maple Syrup Urine Disease
Marshall-Smith Syndrome
Mastocytosis - Type IV
MECP2 Duplication Syndrome
Medulloblastoma - with metastases
Menkes Disease - Classic or Infantile Onset Form
Merkel Cell Carcinoma - with metastases
Merosin Deficient Congenital Muscular Dystrophy
Metachromatic Leukodystrophy (MLD) - Late Infantile
Mitral Valve Atresia
Mixed Dementias
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
Myoclonic Epilepsy with Ragged Red Fibers Syndrome
Neonatal Adrenoleukodystrophy
Nephrogenic Systemic Fibrosis
Neurodegeneration with Brain Iron Accumulation - Types 1 and 2
NFU-1 Mitochondrial Disease
Niemann-Pick Disease (NPD) - Type A
Niemann-Pick Disease-Type C
Nonketotic Hyperglycinemia
Non-Small Cell Lung Cancer
Obliterative Bronchiolitis
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
Pallister-Killian Syndrome
Pancreatic Cancer
Paraneoplastic Pemphigus
Patau Syndrome (Trisomy 13)
Pearson Syndrome
Pelizaeus-Merzbacher Disease-Classic Form
Pelizaeus-Merzbacher Disease-Connatal Form
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