Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...
Why me and not you?’ my twin sister,  Amanda,  asked me.  It was a mild, sunny day in April 2022 and we were at my mum’s ...
A wave of support has emerged online for a Thai child diagnosed with Aromatic L-amino acid decarboxylase deficiency ( AADC Deficiency), a rare genetic disorder affecting only about 130 people ...
An editor in his early 40s thought the tiny numb spot on his nose was innocuous. A year and six doctors later, he learned it ...
One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid increases in genetic technology and testing.
Cushing's syndrome is fairly rare. It most often affects adults who are 20 to 50 years old. It is sometimes called hypercortisolism. You will probably need to see a specialist in hormone-related ...
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
The partners will adapt Eukarÿs' C3P3 engineered enzyme tech to work with adeno-associated virus vectors to boost biomanufacturing yields.
The gene mutation was isolated in a family in northern Italy where under half of the relations developed the disease. View on ...
Surfactant B deficiency causes abnormalities in the lining of the lung tissues, making breathing difficult. The life expectancy without a lung transplant is about three to five months. In Kylie’s case ...
The Phase Ib trial is set to test a gene therapy designed to target a rare disorder that causes muscle and respiratory failure.
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