Gene therapy developments, groundbreaking non-invasive procedures, and interventions on the horizon were the focus of the latest installment of Pennsylvania Hospital’s Franklin-Bond Speaker Series, which offers a multidisciplinary look at PAH’s advances in research, patient care, and preservation. The December session focused on how researchers and clinicians are paving the way for new treatments for movement disorders like Parkinson’s disease and Huntington’s disease, neuromuscular disorders like ALS and Guillain-Barré syndrome, and other related conditions.
At this session, a special guest took to the podium to discuss his experiences receiving exceptional care at PAH: former Pennsylvania Governor Ed Rendell, who announced his Parkinson’s diagnosis publicly in 2018. Rendell shared the impact of his personalized treatment plan that balances medication with physical therapy and exercise. With the support of a care team that offers the best options now while also inspiring hope for future breakthroughs, he has been able to continue leading a full and active life.
An expert panel discussed some of the latest research in the field. For example, PAH is one of the first institutions to offer MR-guided focused ultrasound to patients with essential tremor, and Penn is seeking to identify a biomarker that could aid in early diagnosis of Parkinson’s. Researchers are also developing a gene therapy to treat Duchenne muscular dystrophy; the U.S. Food and Drug Administration’s approval of Zolgensma, a gene therapy that halts the progression of spinal muscle atrophy in children, offers hope for future advances.
“This forum gave us the opportunity to highlight developments that weren’t possible just five years ago, as well to show that Pennsylvania Hospital really treats the whole patient,” said Matthew B. Stern, MD, director emeritus of the Parkinson’s Disease and Movement Disorders Center. “One of our priorities is fostering community engagement, so participating in the Franklin-Bond Speaker Series offered an invaluable opportunity to make those connections.”