News

Register today for the 2022 Clinic for Special Children 5k!

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, and walkers are invited to participate in the Clinic for Special Children 5k – a 3.1-mile course along scenic country roads, winding through Lancaster County […]

Read more

Dr. Kevin Strauss lead author on recently published papers demonstrating the safety & efficacy of SMA gene therapy

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 SMN2 at risk for developing SMA type 1 or 2, respectively. “Two decades following completion of the human genome project, onasemnogene abeparvovec delivers on the […]

Read more

Enjoy our Summer 2022 Newsletter!

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 disorders we see at the Clinic, and what seizures are and appropriate first aid practices. CLICK HERE to read the latest issue now!

Read more

Dr. Kevin Strauss delivers talk at 25th ASGCT Meeting on the safety & efficacy of novel MSUD gene therapy in murine and bovine models

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 gene replacement vector therapy in murine and bovine models of classic Maple Syrup Urine Disease (MSUD). To read a full press release on the talk […]

Read more

Downloadable 2022 CSC Events Calendar

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 compassionate care to children and adults with rare genetic disorders. Click HERE or on the image below to download our 2022 events calendar!

Read more

Enjoy our Spring 2022 Newsletter!

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 Lives Program Manager, and hear a story of the Clinic caring for a neighbor with a terminal rare genetic disease. This newsletter also showcases our […]

Read more

The Effects of NPRL3 Loss Described in New Study in Old Order Mennonite Population

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 localization, and neuron structure. This study reports the largest and genealogically oldest known NPRL3 patient pedigree. The 12-generation, Old Order Mennonite pedigree dates back to […]

Read more

2022 Press Kit Now Available

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. For media inquiries, please contact press@clinicforspecialchildren.org or call us at 717-687-9407.

Read more

Clinic featured on RareShare podcast episode!

  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 within the Plain community and beyond in a podcast episode with RareShare, part of the Rare Genomics Institute. The hour-long podcast episode recaps the Clinic’s […]

Read more

Meet our 2022 Ambassadors!

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 year. We’re thankful to each family for helping us spread the word about the work of the Clinic!   Jevon & Josiah King| 2 years […]

Read more