The early-stage studies being conducted into Sanfilipposyndrome don't administer a drug or treatment.
WSJ: Families Push for New Ways to Research Rare Diseases
2.
Now, another institution, Nationwide Children's Hospital in Columbus, Ohio, will soon launch another study of Sanfilipposyndrome as it works on a potential treatment.
WSJ: Families Push for New Ways to Research Rare Diseases
3.
Ms. Siedman says such a time lag in getting the data out might result in a situation where there are still multiple natural-history studies in Sanfilipposyndrome.
WSJ: Families Push for New Ways to Research Rare Diseases