The Enzyme Replacement Therapy HGT-1110 Phase I/II clinical trial has been fully recruited. Patients are participating from three continents; Europe, South America, and Australia. The primary purpose of a Phase I/II clinical trial is to study safety and to establish a target dosage for further study. The trial lasts 40-weeks so the last patient should complete their […]
ERT Phase I/II Clinical Trial Recruiting Completed Read More »
Great Patient Focused Drug Development (PFDD) meeting at the FDA Tuesday discussing patients perspective on the neurological inborn errors of metabolism (IEM). Dean Suhr, president of the MLD Foundation, spoke on the second panel of the day and was able to both share and stir the pot a bit with regard to some of the patient perspectives on
We are pleased to share that a Phase I/II Intracerebral Gene Therapy clinical trial for MLD is now recruiting late infantile MLD patients. Dr. Patrick Aubourg and Dr. Caroline Sevin are the co-Principal Investigators. We have posted complete details of the trial, including inclusion criteria, here. This trial is based on many years of work in
Intracerebral Gene Therapy Phase I/II Clinical Trial for MLD Read More »
