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Drug Pipeline
Sys. Review
Mini-review discusses ATM protein role in neural stem progenitor cells in ataxia telangiectasia
How a Brain’s “Master Switch” Controls Child Brain Development
This minireview examines the function of ATM protein in neural stem progenitor cells (NSPC) within the context of ataxia telangiectasia. The…
Understanding how ATM protein guides brain cell growth could lead to better treatments for a rare childhood brain disorder.
Frontiers
Apr 19, 2026
Hematology
Meta-analysis
Meta-analysis of HSCT with treosulfan in Ataxia Telangiectasia patients shows mixed survival outcomes and significant toxicities.
A Teen With a Rare Disease Beat Leukemia After a Risky Transplant
This systematic review and meta-analysis evaluated hematopoietic stem cell transplantation (HSCT) using treosulfan in 16 patients with Ataxi…
A 16-year-old with a rare genetic disease survived leukemia after a tailored stem cell transplant, offering new hope for others with this fr…
Frontiers
Apr 16, 2026