STRASBURG, PA & TOPEKA, IN- Researchers and clinicians through a multicenter collaboration have identified a novel multisystem disorder caused by bi-allelic variants in the CCDC47 gene. Their findings are reported […]

On Friday, November 16, 2018 over 210 Extraordinary people made a donation to the Clinic for Special Children with over $62,000 raised in just 24 hours! The community shattered our […]

STRASBURG, PA- Researchers have identified a novel missense mutation in tyrosyl-tRNA synthetase (YARS c.499C>A, p.Pro167Thr) that causes a severe recessive disorder in affected individuals. The study, led by clinicians, researchers […]

In this edition of the CSC newsletter, read about the Watson family’s journey with GA-1, our partnership with the Strasburg Rail Road, new CSC staff members, recent research study updates, […]

THANK YOU to everyone who came out on Saturday, September 22nd and made our first Clinic for Special Children 5k such a success! Because of you and over 280 registrants, […]

STRASBURG, PA- A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy (SMA) within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes […]

STRASBURG, PA- A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting […]

Please join us for our FIRST 5K Run/Jog/Walk to benefit Clinic for Special Children! $100 CASH PRIZE for overall top male & female runners! Enjoy a 5K, free refreshments, a […]

  In this edition of the CSC newsletter, read about the Martin family’s inspiring journey with Kleefstra Syndrome. Also in this issue is the story of Margaretha whom the Clinic […]

STRASBURG, PA- A new comprehensive natural history study about Amish nemaline myopathy (ANM) in the Old Order Amish population focuses on the promise of gene therapy for this lethal disorder. […]