NUCDF announces its 2024 Medical Advisory BoardNUCDF is pleased to announce the members of its 2024 Medical Advisory Board, which advises its leadership on key topics like new research discoveries and new medicines or treatments related to urea cycle disorders. The 2024 board includes:
- Nicholas Ah Mew, M.D., Children’s National Hospital, Washington, D.C
- James A. Bartley, M.D., Loma Linda University School of Medicine
- Susan A. Berry, M.D., Department of Pediatrics, University of Minnesota
- Lindsay Burrage, M.D., Ph.D., Baylor College of Medicine
- Andrea L. Gropman, M.D., FAAP, FACMG, FANA, Children's National Hospital, Washington, D.C.
- Brendan Lee, M.D., Ph.D., Baylor College of Medicine
- George Mazariegos, M.D., University of Pittsburgh School of Medicine
- Sandesh C.S. Nagamani, M.D., FACMG, Baylor College of Medicine
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| Sponsor a floating lantern tribute for Cindy Le Mons
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Can't attend the upcoming NUCDF Family Conference? You can still honor our former director, Cindy Le Mons, by sponsoring a floating lantern. The lanterns will be released during our Friday, April 5, evening memorial service as a tribute to her decades of work on behalf of the UCD community.
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mRNA technology used to correct arginosuccinic aciduria in a mouse model
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By exploiting the technology used in Covid-19 vaccines, a team led by University College London, King’s College London, and Moderna scientists found that messenger RNA (mRNA) could be used to correct the rare liver genetic disease argininosuccinic aciduria (also known as arginosuccinate lyase deficiency or ASLD) in a mouse model of the disease.
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Researchers document father-to-daughter transmission in late-onset OTC deficiency
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A research team exploring the inheritance patterns of late-onset ornithine transcarbamylase deficiency (OTC deficiency) has described two Caucasian families showing paternal transmission of the disorder. Previously, father-to-daughter OTC deficiency transmission had only been documented in four Japanese families.
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