Peer-Reviewed Publication MURDOCH CHILDRENS RESEARCH INSTITUTE A drug that boosts bone growth in children with the most common form of dwarfism, may also reduce their chances of sudden infant death syndrome, sleep apnoea and needing surgery, according to a new study. The international research trial, led by Murdoch Children’s Research Institute (MCRI) and published in...
Tag: <span>dwarfism</span>
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Promising results shown in the treatment of growth-hormone resistant dwarfism
With gene therapy, a cure for growth-hormone resistant dwarfism may be on the horizon. NATIONAL UNIVERSITY OF SINGAPORE, YONG LOO LIN SCHOOL OF MEDICINE Researchers from the NUS Yong Loo Lin School of Medicine (NUS Medicine) have shown that gene therapy using a single-dose injection of a virus carrying the ‘good’ gene can potentially be...
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FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism
Jean-Jacques Bienaimé (BioMarin via Youtube) November 19, 2021 11:10 AM EST FDA+ FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism. The FDA on Friday signed off on an accelerated approval for Biomarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics...