Nager syndrome (NS) is an extremely rare disease that causes developmental problems and anomalies in facial bone structures and limbs. While the causative gene is known, its underlying mechanisms ...
NUZ-001 and its active metabolite NUZ-001 Sulfone demonstrated significant neuroprotective effects in a zebrafish model of Huntington's disease Treatment prevented hallmark developmental and ...
BIRMINGHAM, Ala. – Can a small fish help identify possible treatments for an ultra-rare inherited disease found in an Alabama boy? The genetic disease is XMEA, which progressively weakens the muscles ...
A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...
Nearly one million people in the United States are living with Parkinson's disease, making it the second-most common neurodegenerative disease after Alzheimer's. Current medical treatments for ...
The joint project began when a nine-year-old boy came to Prof. Shoshana Greenberger’s clinic at Sheba’s Safra Children’s Hospital with severe shortness of breath and was diagnosed with KLA. Seeking to ...
NUZ-001 and its active metabolite NUZ-001 Sulfone demonstrated significant neuroprotective effects in a zebrafish model of Huntington's disease Treatment prevented hallmark developmental and ...
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