What Is the Life Expectancy of Someone With Sanfilippo Syndrome?
Medicinenet,
The majority of patients with Sanfilippo syndrome do not live past the age of adolescence; however, some people may live longer…
The majority of patients with Sanfilippo syndrome do not live past the age of adolescence; however, some people may live longer…
Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a rare neurodegenerative disease that first appears in…
BOSTON - (Nasdaq: ORTX), a global gene therapy leader, yesterday reported initial data from an ongoing proof-of-concept…
Preliminary results from first patient treated with OTL-201 show over expression of the therapeutic enzyme and decrease in…