UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington / D.C. / U.S. Food & Drug Administration / European Medicines Agency / U.S. Food and Drug Administration / National Organization for Rare Disorders / I.R.S. / European Union / Securities and Exchange Commission / Ministry of Health / /
Person
MPS IIIB / / /
Position
independent investigator / physician / Principal Accountant / Director Independence / focused physician / Sanj K. Patel President and Chief Executive Officer / Principal Executive / /
Product
Sebelipase Alfa / other investigational new drug / Japan / EMA / Animal Drug / /
ProvinceOrState
Delaware / Georgia / Massachusetts / /
PublishedMedium
Natural History / /
Region
Asia Pacific / /
Technology
alpha / antibodies / transplantation / production technology / two clinical study protocol / drug development / /