Gene Therapy for X-linked Severe Combined Immunodeficiency

计划入组 5 人 · 首次发布于 2011 年 8 月 4 日

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

计划入组 1 人 · 首次发布于 2010 年 8 月 4 日

Gene Therapy for X Linked Severe Combined Immunodeficiency

计划入组 10 人 · 首次发布于 2020 年 2 月 27 日

stem cell gene therapy (X-linked severe combined immunodeficiency)

Firolimogene autotemcel

RAG1 CRISPR/Cas9 gene therapy (severe combined immunodeficiency disease)

COMBINED MILITARY HOSPITAL

Pakistan

Molecule X LLC

Mexico

SFJ Pharmaceuticals X, LTD.

Gene Therapy Breakthrough Effectively Cures 'Bubble Boy' Disease in Infants

Gene Therapy for X-SCID Reveals Critical Role of Virtual Memory T Cells in Early Human Immunity

FDA Grants Orphan Drug and Rare Pediatric Disease Designations to ATSN-201 for X-Linked Retinoschisis