Table 1.

Summary of studies conducted on development of therapies for Sanfilippo disease (MPS III) and reported during last 3 years (2014–2017)

MPS III subtype	Models or trials	Therapies investigated in recent years
		ERT	SRT	Other small molecules	Gene therapy
MPS IIIA	Cellular models	X	X	X
	Animal models	X			X
	Clinical trials	X	X		X
MPS IIIB	Cellular models	X	X	X
	Animal models	X	X		X
	Clinical trials		X		X
MPS IIIC	Cellular models		X	X
	Animal models
	Clinical trials		X
MPS IIID	Cellular models
	Animal models				X
	Clinical trials

The availability of reports on particular kinds of therapies and models are marked by X

Abbreviations: MPS mucopolysaccharidosis, ERT enzyme replacement therapy, SRT substrate reduction therapy