REGISTER TODAY! Join us for the 5th annual Clinic for Special Children 5k on Saturday, September 17th from 9:00 a.m. – 11:00 a.m presented by Nemours Children’s Health. Runners, joggers, […]

Recently published companion papers detail the safety and efficacy of onasemnogene abeparvovec, a gene replacement therapy for spinal muscular atrophy (SMA), for presymptomatic infants with two or three copies of […]

Our summer newsletter is hot off the press!  This summer’s newsletter focuses on seizure disorders that we commonly see at the Clinic. Read about a family’s journey with DEPDC5, common seizure […]

Dr. Kevin Strauss delivered a podium presentation on Tuesday, May 17th at the annual ASGCT meeting in Washington D.C. The presentation detailed the safety and efficacy results of a dual-function […]

From auctions to a 5k race, there’s plenty of fun to be had at our upcoming 2022 CSC events! All events benefit the Clinic’s mission of providing accessible, affordable, and […]

Our first newsletter of the year is here! The Spring 2022 newsletter focuses on our palliative care program, Cherished Lives. Learn about palliative care, read an interview with our Cherished […]

STRASBURG, PA – A new study published in this month’s issue of Brain details the effects of the loss of gene products from NPRL3 on seizure threshold, cortical lamination, mTOR […]

The newest version of our press kit is now available here! Meet our 2022 ambassador families and learn more about how we practice translational medicine and research here at the Clinic. […]

  RareShare Podcast Episode | February 2022 Dr. Kevin Strauss, Medical Director, Dr. Erik Puffenberger, Laboratory Director, and Karlla Brigatti, MS, CGC, Research Operations Director, shared about the Clinic’s work […]

We’re excited to introduce our 2022 Clinic for Special Children ambassadors! Our ambassadors and their families will tell their stories and represent the Clinic in a variety of ways this […]