Month: June 2022

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

Posted on by Kelly Cullen

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 farmland, venturing past a vineyard, an Amish schoolhouse, and acres of picturesque farms. Strollers and wheelchairs are welcome!

Click on the links below to register online or via a paper application.

 

 

 

WHERE | The manually-timed course starts and ends at the Clinic for Special Children, 535 Bunker Hill Road, Strasburg, PA 17579.

TIMES | 9:00 a.m. start for 5k race. Kid’s color fun run following the main 5k race. Race day registration for the 5k race & kid’s color fun run will open at 7:45 a.m. and close promptly at 8:45 a.m.

PACKET PICK-UP | Friday, September 16 between 9 a.m. – 5 p.m. and race day from 7:45 a.m. – 8:45 a.m. at the Clinic for Special Children.

5K ENTRY FEES
$30 | by August 19, includes t-shirt (entry & payment must be received by Aug. 19).
$35 | after August 19 (including race day) t-shirt while supplies last.
$10 | Kid’s color fun run for ages 10 & under. Kid’s color fun run to start after conclusion of main 5k race.

HOW TO REGISTER
1. You can register online HERE at Pretzel City Sports. Online registration has a nominal service fee and closes at midnight Weds. 9/14.
2. Mail registration form & payment to: Clinic for Special Children, Attn: 5k Registration, PO Box 128, Strasburg, PA 17579. If paying via check, make payable to: Clinic for Special Children
3. Register in-person on race day. Race day registration will be open at 7:45 a.m. and close promptly at 8:45 a.m.

5k AWARDS | Medals will be awarded to the 1st overall male and female finishers. Awards will be given to the top three male and female finishers in the following categories: 14 & under, 15-19, 20-29, 30-39, 40-49, 50-59, 60-69, 70+.

QUESTIONS | Contact us at queries@clinicforspecialchildren.org or call 717-687-9407.

Event will be held rain or shine. No refunds, mailed awards or t-shirts. Results posted on PretzelCitySports.com & photos posted Mon., Sept. 19 on ClinicforSpecialChildren.org. Events are subject to change due to severe weather conditions, restrictions, or unforeseen circumstances. Visit www.ClinicforSpecialChildren.org/events/5k for updates.

 

Thank you to our sponsors!

Presenting Sponsor

Kid’s Fun Run Sponsor

Platinum Sponsors

Gold Sponsors

Eberly Plumbing

Silver Sponsors

Dienner Home Improvement

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

Posted on by Kelly Cullen

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 promise of that great undertaking, demonstrating the transformative potential of gene-based therapies for previously intractable hereditary disorders in children. SPR1NT provides an example of what can be achieved when newborn screening is combined with safe and effective disease-modifying therapy. It represents remarkable evolution in the SMA standard of care, from a reactive to a proactive stance; from a focus on patients who survive to children who thrive. At the Clinic for Special Children, we are committed to applying this preemptive model to other life-threatening conditions enriched in the populations we serve, and deeply value the community’s partnership in that endeavor,” according to Dr. Strauss, lead author on both papers.

Together, these papers summarize the final results of SPR1NT, a Phase III study focusing on newborns administered one-time intravenous SMN gene replacement therapy before six weeks of age. SMA is a devastating genetic disorder that leads to progressive degeneration of spinal motor neurons that control movement, swallowing, and breathing. Untreated infants with SMA Type 1 do not achieve independent sitting or other advanced motor milestones and 100% die or require permanent ventilation by two years of age. Infants with untreated SMA Type 2 sit independently but do not walk, and develop debilitating musculoskeletal and respiratory complications with advancing age. The companion papers, divided into SMN2 two-copy (n=14) and three-copy (n=15) cohorts, were recently published in the journal Nature Medicine. Dr. Kevin A. Strauss, Medical Director at the Clinic for Special Children, served as first author.

To read the SMN2 two-copy paper, click HERE. 

To read the SMN2 three-copy paper, click HERE. 

To read the full press release, click HERE.