Maria Escolar, MD, on Combining HSCT With Gene Therapy in Krabbe Disease
The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.
Maria Escolar, MD, on Addressing Unmet Needs in Krabbe Disease With Gene Therapy
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
Patients With Fabry Disease Withdrawn From ERT After Isaralgagene Civaparvovec Treatment
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.