MPS (RaDiCo Cohort) (RaDiCo-MPS)

计划入组 1,000 人 · 首次发布于 2023 年 9 月 14 日

Oral or Intra Muscular Vitamin D2 Vs Oral or Intra Muscular Vitamin D3 in Treatment of Vitamin D Deficiency

计划入组 100 人 · 首次发布于 2024 年 9 月 30 日

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

计划入组 96 人 · 首次发布于 2003 年 10 月 1 日

lentivirus-based gene therapy (multiple sulfatase deficiency)

AAV9-based gene therapy (multiple sulfatase deficiency)

Cerebroside sulfatase

Multiple Myeloma Research Consortium

United States

National Multiple Sclerosis Society

United States

MULTIPLE MYELOMA RESEARCH FOUNDATION

United States

FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies

JCR Pharmaceuticals' JR-441 Receives Clearance for Phase I MPS IIIA Trial in Japan

FDA Extends Review Timeline for REGENXBIO's Hunter Syndrome Gene Therapy RGX-121