Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

计划入组 5 人 · 首次发布于 2025 年 4 月 28 日

Cognitive Disorders in Hereditary Spastic Paraplegia Type 4

计划入组 30 人 · 首次发布于 2024 年 2 月 15 日

Physiotherapy in Hereditary Spastic Paraplegia

计划入组 53 人 · 首次发布于 2019 年 5 月 23 日

AAV9 vector-based AP4B1 gene therapy (hereditary spastic paraplegia 47)

AP4M1 gene therapy (AAV9, hereditary spastic paraplegia 50)

chPcMab-47

Cipla Limited

India

海南如康生物科技有限公司

Q Therapeutics, Inc.

United States

FDA Clears BlackfinBio's Gene Therapy Trial for Rare Hereditary Spastic Paraplegia

FDA Clears BlackfinBio's Novel Gene Therapy Trial for Rare Childhood Neurological Disease SPG47

CD99L2 Identified as Novel Disease Gene for Spastic Ataxia and Hereditary Spastic Paraplegia