Tag: Gene Therapy
FDA Approves First Gene Therapy for Spinal Muscular Atrophy
Patients treated with Zolgensma have shown improvement in reaching developmental motor milestones
Gene Therapy Forms New Polysynaptic Pathways in Parkinson’s
Patients receiving GAD gene therapy develop unique treatment-dependent polysynaptic brain circuit
Potential First U.S. Gene Therapy Now Under FDA Review
Tisagenlecleucel could be a one-time treatment for children and young adults with advanced leukemia
Gene Tx May Benefit Children, Teens With Wiskott-Aldrich Sx
Patient's own blood stem cells are removed to correct the WAS gene, then injected back into the patient
