Programs

PNTC programs bring together cutting-edge research, expert clinical care, and patient-centered support to advance therapies for neurodegenerative diseases. Through collaborative, multidisciplinary initiatives, we are working to accelerate discoveries, expand access to innovative treatments, and improve outcomes for individuals and families affected by neurogenetic conditions.

The Penn ALS Program unites clinicians and researchers in a collaborative effort to advance therapeutic development and improve clinical care for individuals living with amyotrophic lateral sclerosis (ALS).

Our Penn ALS Program webpage is currently under development.

This program is focused on advancing individualized genetic medicine approaches for patients with ultra-rare neurologic diseases. Bringing together clinicians, researchers, genetic counselors, and translational experts across Penn and CHOP, the program explores pathways for developing personalized N=1 therapies, including emerging gene-targeted technologies.

PEN[N=1] is collaboratively evaluating infrastructure needs and potential candidate programs for future therapeutic development. Additional information and program updates will be shared as the initiative evolves.

If you or a family member has been diagnosed with an ultra-rare neurogenetic disease and you are interested in learning more about an individualized therapeutic strategy, please contact us at NeurogeneticsTherapyCenter@pennmedicine.upenn.edu.

At present, we are in the early stages of developing a registry that will facilitate enumeration of interested patients, establishment of a biobank of samples and cell lines, and collaboration for therapeutic development.

Our PEN[N=1] webpage is currently under development.