Lysosomal Disorders

Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion

The positive opinion was based on findings from studies conducted in Taiwan.

Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene Therapy in PKD

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

Gene Therapy for LAD-I Posts Impressive Survival Data

In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.