Meet baby Fitz Baby Fitz was born with a dysfunctioning immune system. His treatment offers hope for curing rare diseases. Fitz Kettler was born June 21, 2019, without a functioning immune system. Babies with his condition, commonly known as “bubble boy...
A new way to find genetic variations removes bias from human genotyping Since the first international effort to sequence the 3 billion DNA letters in the human genome (The Human Genome Project), the study of human genomes has relied almost exclusively on a...
Access inequality for European children to newborn screening Children across Europe are facing early death or lifelong disability because of a lack of access to disease diagnosis through existing newborn screening (NBS) programs. Now is the time to ensure all...
New gene therapy approach offers a potential long-term treatment for limb-girdle muscular dystrophy 2B. Children’s National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B,...
As the end of another challenging year draws to a close, we are reflecting on everything we have achieved and setting ambitious goals for the year to come. Fuelled in large part by the coronavirus pandemic, 2021 brought new challenges no one was prepared for,...
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