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Distribution of respondents (n = 181) by patient advocacy groups
| Patient group | N (%) |
|---|---|
| National Mucopolysaccharidosis Society (USA) | 100 (55.2%) |
| United Kingdom Mucopolysaccharidosis Society | 36 (19.9%) |
| Duke Pompe Clinical and Research Group (USA) | 24 (13.3%) |
| International Gaucher Alliance | 12 (6.6%) |
| Instituto Vidas Raras (Brazil) | 9 (5%) |
