Announcements & News
Stay up-to-date on important announcements and news! The most recent announcements and news are located on top. Please click on the toggle of each item to reveal more information.
Announcements
New Clinical Staff
The Penn HD Center of Excellence is thrilled to announce that we have a new neurologist on our team:
Dr. Stephen Aradi joined Penn Neurology in 2026 as an Assistant Professor of Clinical Neurology. He completed his neurology residency at the University of Pennsylvania in 2019, followed by fellowship training in movement disorders with a focus on Huntington disease at the University of South Florida in 2020. Prior to joining Penn Neurology he served as the Director of the HDSA Center of Excellence at USF, where he gained experience as principal investigator of several observational and interventional clinical trials in HD. He is passionate about multidisciplinary care for complex neurological disorders.
The Penn HD Center of Excellence is proud to welcome Dr.Laura Duque, MD as our new neuropsychiatrist. She is succeeding Dr.Alexander Zwil, MD in this position. Please see her biography on our team homepage.
We are also pleased to welcome Katie, our therapy dog. Katie will be present each clinic day and is a resource for patients, family members, and staff.
Novel Therapy for HD
The Penn HD Center of Excellence team is excited by the positive top-line results from uniQure's Phase I/II trial of AMT-130.
AMT-130 is a one-time gene therapy administered via brain surgery to treat Huntington's Disease. This news, announced September 24th, 2025, is extremely promising for individuals and families living with HD.
The top-line results from the study show that symptom progression is being significantly slowed by the drug. This is the first time any drug has been shown to alter the course of HD in a clinical trial.
The idea behind AMT-130 is to reduce the amount of huntingtin protein that cells produce. AMT-130 is the first gene therapy designed specifically for HD that has made it into human clinical trials. Instead of being taken as a pill or an injection, AMT-130 is delivered directly into the brain through a surgical procedure.
Some important factors to note about these results are that the trial had fewer than 30 participants, only a portion of whom received the high dose of the drug, which shows the most benefit. Only subjects with early-symptomatic disease were included, although additional trials have been announced with different populations.
For our patients here at Penn, we especially want to note that while several sites in the US are involved in this study, this treatment has not yet been approved by the FDA and is not yet available for clinical use.
uniQure reportedly plans to meet with the FDA later this year to discuss the data. We will have to wait to see how the FDA responds. The Penn HD Center of Excellence and our HD research team will aim to keep our community updated on the progress of this and other encouraging trials.
Links for Further Information:
Researchers say new therapy may slow Huntington’s disease Dr.Lasker was interviewed by KYW Radio- listen here!
Press Releases | Investors & Media | uniQure
The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial – HDBuzz
***The FDA gave the Pfizer-BioNTech COVID-19 vaccine authorization to use in children ages 5 and older.*** The F.D.A. broadened Pfizer’s authorization to cover the younger age group (age 5-15 years old) on an emergency basis. The Moderna and Johnson & Johnson vaccines remain authorized only for adults.
Since December 9, 2021, Federal regulators authorized booster shots of the Pfizer-BioNTech coronavirus vaccine for another swath of the population, 16- and 17-year-olds, at least six months after they received their second shot of that vaccine. Everyone 18 and older has been eligible since Nov. 19, 2021 to receive a booster, six months after getting a second shot of the Pfizer or Moderna vaccine, or two months after a Johnson & Johnson shot.
In a nutshell: If you are offered a vaccine by your health care provider, we recommend that you receive the vaccine as offered unless there is a reason you should avoid that vaccine, such as an allergy to an ingredient (click here for more information). Inform your vaccination provider about all your allergies and health conditions.
Please click on the title of this announcement for more information.
Vaccine Information
If you would like to schedule a COVID vaccine appointment at Penn Medicine, please call the vaccine scheduling hotline at 267-758-4902. They answer calls between 8:00 am - 5:00 pm on Monday through Friday. The vaccines are NOT being administered at Penn's HD Center, but they are available at other Penn Medicine locations. View locations and hours information on the Penn Medicine vaccine website.
If you would like us to keep you informed about COVID vaccine efforts at Penn Medicine, please complete the Penn Medicine Vaccine Interest Form (this form will NOT sign you up for a vaccine appointment, but it will keep you informed on the latest vaccine news).
*For more information about vaccines and HD, please click here to view an article by HDBuzz.*
*For general vaccine questions, please click here.*
How to find other vaccine providers near you:
- Visit Vaccines.gov to find vaccination providers near you. In some states, information may be limited while more U.S. vaccination providers and pharmacies are being added. Learn more about COVID-19 Vaccination Locations on Vaccines.gov.
- Text your ZIP code to 438829 or call 1-800-232-0233 to find vaccine locations near you in the U.S.
- Check your local pharmacy’s website to see if vaccination appointments are available. Find out which pharmacies are participating in the Federal Retail Pharmacy Program.
- Contact your state health department to find additional vaccination locations in the area.
- Check your local news outlets. They may have information on how to get a vaccination appointment.
More information by state:
Vaccine Safety
To date, there is no indication that these vaccines pose any added risk to patients with Huntington's disease or related disorders compared to the general population. There is also no interaction between the vaccine and Huntington's disease medications.
If you are offered a vaccine by your health care provider, we recommend that you receive the vaccine as offered unless there is a reason you should avoid that vaccine, such as an allergy to an ingredient (click here for more information). Inform your vaccination provider about all your allergies and health conditions.