Register today for the 2022 Clinic for Special Children 5k!
Posted on June 20, 2022
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, […]
Dr. Kevin Strauss lead author on recently published papers demonstrating the safety & efficacy of SMA gene therapy
Posted on June 17, 2022
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 […]
Enjoy our Summer 2022 Newsletter!
Posted on June 14, 2022
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 delivers talk at 25th ASGCT Meeting on the safety & efficacy of novel MSUD gene therapy in murine and bovine models
Posted on May 18, 2022
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 […]
Downloadable 2022 CSC Events Calendar
Posted on March 30, 2022
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 […]
Enjoy our Spring 2022 Newsletter!
Posted on March 24, 2022
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 […]
The Effects of NPRL3 Loss Described in New Study in Old Order Mennonite Population
Posted on March 14, 2022
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 […]
2022 Press Kit Now Available
Posted on March 11, 2022
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. […]
Clinic featured on RareShare podcast episode!
Posted on February 8, 2022
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 […]
Meet our 2022 Ambassadors!
Posted on February 3, 2022
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 […]