Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

计划入组 30 人 · 首次发布于 2023 年 4 月 24 日

Natural History to Assess Disease in Patients With MPS IIIC

计划入组 35 人 · 首次发布于 2026 年 7 月 17 日

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

计划入组 4 人 · 首次发布于 2011 年 11 月 18 日

stem cell based gene therapy (Sanfilippo syndrome type B)

NAGLU gene AAV9 therapy (sanfilippo syndrome B)

gene therapy (adeno-associated virus, Sanfilippo syndrome)

Sanfilippo Therapeutics SAS

ALPORT SYNDROME FOUNDATION

United States

Abeona Therapeutics

United States

Phoenix Nest Secures $1.49 Million NIH Grant to Advance Gene Therapy Manufacturing for Sanfilippo Syndrome Type C

Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A

FDA Approves Fayuvi, First-Ever Gene Therapy for Sanfilippo Syndrome Type A