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Neuromuscular
Duchenne Muscular Dystrophy
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Hereditary Transthyretin (hATTR) Amyloidosis
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Central nervous system
AADC Deficiency
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Friedreich’s Ataxia
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Mitochondrial Disease Associated Seizures (MDAS)
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Huntington’s Disease
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Angelman Syndrome
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Amyotrophic lateral sclerosis (ALS)
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Metabolism
Familial Chylomicronemia Syndrome (FCS)
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Phenylketonuria (PKU)
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Oncology
DIPG
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LMS
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Across Oncology
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