Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Pyruvate Kinase Deficiency and Severe Leukocyte ...
Sustained genetic and phenotypic correction and concomitant hematologic stabilization observed in at least six of 10 evaluable FA patients between 12 and 36 months after RP-L102; a seventh patient potentially demonstrates engraftment and stabilization …