Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study

Table 4 Therapy received and location of weekly infusion of ERT

	Group A	Group B	Group C
Therapy received	(n_p = 8)	(n_p = 10)	(n_p = 7)
ERT	4	8	6
HSCT	2	2	1
ERT + HSCT	2	–	–
Location of therapy	(n_p = 6)	(n_p = 8)	(n_p = 7)
ERT received in Home Hospital Care	2	3	–
ERT received in Day Hospital	4	5	7

n_p, Number of patients
ERT Enzyme replacement therapy, HSCT Haematopoietic stem cell transplantation
Group A, Patients not able to answer the survey question because of a severe cognitive impairment (n_p = 8); Group B, Patients able to answer the survey question with low or no cognitive impairment and high motor disability (n_p = 10); and Group C, Patients able to answer the survey question with low or no cognitive impairment and low motor disability (n_p = 7)

ISSN: 1750-1172