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Group by: No Grouping | Volume Number of items: 2. 10Weinreb, NJ and Finegold, DN and Feingold, E and Zeng, Z and Rosenbloom, BE and Shankar, SP and Amato, D (2015) Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3). Orphanet Journal of Rare Diseases, 10 (1). 7Grünert, SC and Wendel, U and Lindner, M and Leichsenring, M and Schwab, KO and Vockley, J and Lehnert, W and Ensenauer, R (2012) Clinical and neurocognitive outcome in symptomatic isovaleric acidemia. Orphanet Journal of Rare Diseases, 7 (1). |
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