MeiraGTx receives FDA Rare Pediatric Disease Designation for AAV8-RK-RetGC to treat Leber congenital amaurosis due to GUCY2D mutations. MeiraGTx Holdings Plc announced that the FDA has granted its ...

"This expansion marks the culmination of years of dedicated effort," said Dr. Frank Scholl, Chief of Joe DiMaggio Children's Hospital Heart Institute and Pediatric & Congenital Cardiac Surgery.

The FDA approval of crinecerfont (Crenessity; Neurocrine Biosciences) on December 13, 2024, marked a significant milestone in the management of classic congenital adrenal hyperplasia (CAH), ...

The Adult Congenital and Pediatric Cardiology Member Section is only able to accomplish its mission through support from members. Members can join online by clicking here or by contacting ACC Member ...

it will allow swift intervention as children need volume for volume blood transfusion in case of blood loss or emergency surgery,” the researcher on congenital pediatric oncology said.

Pediatric foot deformity is a term that includes a range ... The primary symptom from this additional bony prominence is pain and tenderness. This congenital defect (present at birth) is thought to ...

The American Academy of Pediatrics (AAP) has recently updated screening recommendations for newborn critical congenital heart disease (CCHD). The birth defect causes the highest number of deaths among ...

emirates.net.ae Dyskeratosis congenita (DC) is a rare inherited multisystem disorder characterised by lesions of the skin and appendages. Bone marrow failure occurs in 80% of patients. The gene for ...

Seattle Children’s pediatric orthopedics team partners with researchers around the world to make sure we are using the latest treatment approaches and technologies. It’s part of our commitment to ...

1 Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland, USA 2 ...