Table 1.

Summary of patients with Morquio A

	Case 1	Case 2	Case 3	Case 4
Gender	F	M	M	M
Present age	25 years	36 years	31 years	26 years
Diagnosis	1.5 years	4 years	12 years	14 years
Age at transplantation	4 years	8 years	15 years	15 years
Follow up duration	21 years	28 years	16 years	11 years
Regimen of BMT	busulfan (20 mg/kg ), cyclophosphamide (200 mg/kg), anti T- lymphocytic globulin (10 mg/kg/day)	cyclophosphamide (200 mg/kg), thoracoabdominal irradiation 8 Gy	Detailed data not avialble (Performed at Nippon Medical School)	busulfan (4 mg/kg), cyclophosphamide (50 mg/kg), anti T-lymphocytic globulin (15 mg/kg/day)
Genotype	p.M318R/undefined	Double deletion/p.Q148X	ins ex1/undefined	Undefined
Donor	HLA-identical sibling	HLA-identical unrelated donor	HLA-identical heterozygous sibling	HLA-identical heterozygous sibling
GVHD	grade I	grade I	None	None
Engraftment	Full	Full	Full	Full
Enzyme activity	Normal	Normal	carrier level	carrier level
Walking	walk independently	walk independently	walk independently	walk independently
Height	113 cm at 20 years	115 cm at 24 years	137 cm at 18 years	103 cm at 25 years
ADL score (full score 60: higher is better)	59	33	55	34 to 46*
Other MPS Symptoms (full score 60: higher is better)	50	42.5	51	44 to 56*
Orthopedic Surgical Intervention post BMT	ND	ND	ND	Osteotomies of both femurs at 16 years

ND: not done

^*pre-BMT to post-BMT