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https://doi.org/10.1007/978-0-387-47672-8LSDs that have central nervous system (CNS) involvement, where enzyme replacement therapy (ERT), the standard of care for LSDs, is ineffective in treating the neurological features of these diseases. Recombinant adeno-associated virus (AAV) vectors have emerged as the most efficient and promising ge

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Jochen Axer improvement included improved prophylaxis of opportunistic infections, especially.pneumonia (PCP), and improved recognition and management of other opportunistic infections. Although PCP prophylaxis led to a reduction in deaths due to PCP, it was also associated with an increased risk of wasting sy

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