"Childhood" and "dementia" are two words we wish we didn't have to use together. But sadly, around 1,400 Australian children and young people live with currently untreatable childhood dementia.
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The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.
Oct 30, 2023
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Using a protocol developed at UC San Francisco, physicians have successfully treated a fetus with a devastating genetic disorder for the first time, and the child is now thriving as a toddler, a case study in the New England ...
Nov 10, 2022
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The U.S. Food and Drug Administration has approved Xenpozyme (olipudase alfa) for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD), a rare genetic disease.
Sep 5, 2022
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Autoimmune conditions like Type 1 diabetes, multiple sclerosis and rheumatoid arthritis arise when an immune system mistakenly attacks its own body's proteins, cells and organs. Not only do these conditions make the body ...
Nov 29, 2021
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Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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(HealthDay)—Nexviazyme (avalglucosidase alfa-ngpt) was approved for the treatment of the rare inherited disorder late-onset Pompe disease in patients 1 year of age and older, the U.S. Food and Drug Administration announced ...
Aug 11, 2021
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An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
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Rare diseases are sometimes the most difficult to treat because of a lack of research and fewer participants to study.
Apr 16, 2021
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A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia ...
Feb 24, 2021
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