An investigational oral treatment aimed at a common link to Parkinson's disease recently began undergoing a phase II trial, the drug's maker, Sanofi Genzyme, announced recently.
The trial includes Parkinson's patients who carry a single gene copy mutation — GBA — that is most commonly linked with the disease. The study will evaluate the drug's efficacy, dynamics and safety.
“Patients with Parkinson’s disease and a GBA gene mutation are predisposed to develop motor symptoms at a younger age, have a higher prevalence of cognitive impairment and undergo more rapid disease progression,” Dr. Anthony Schapira, lead investigator of the study, said.
Sanofi, the parent company of Sanofi Genzyme, said that investigating a more targeted therapy approach for these specific patients could be a first step in meeting the needs of all those managing Parkinson's disease.
Parkinson’s is a chronic, degenerative neurological disorder that affects approximately 5 million people, including approximately 1 million Americans. Five percent to 10 percent of those with the disease carry the gene mutation under evaluation.
