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Meet the NIH Detectives Cracking Medicine's Toughest Cases

Meet the NIH Detectives Cracking Medicine's Toughest Cases
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United States , Far Side , Maria Acosta , David Adams , Graham Tucker , William Gahlis , Lynne Wolfe , Francis Rossignol , William Gahl , Don Hadley , Cynthia Tifft , Ellen Macnamara , Anna Savvidis , Stephen Groft , Camilo Toro , Special Olympics , University Of Wisconsin , National Tay Sachs , Office Of Rare Disease Research , Diseases Association , Clinical Center , National Human Genome Research Institute , Kyoto University , National Institute Of Child Health , Human Development , Diseases Program ,

Potentially Protective Agent Shows Benefit in Niemann-Pick Type C

Better neurologic symptoms after treatment with N-acetyl-L-leucine ....

United States , Cynthia Tifft , Sophie Putka , National Human Genome Research Institute In Bethesda , England Journal , National Human Genome Research Institute , Wall Street Journal , Business Insider , Cannabis Wire , Medpage Today , Englj Med ,

Baylor scientists link MRTFB gene variants with novel neurodevelopmental disorder

Researchers at Baylor College of Medicine have linked specific variants or mutations of the gene myocardin-related transcription factor B (MRTFB) with a novel neurodevelopmental disorder. ....

United States , Shinya Yamamoto , Ellenf Macnamara , Cynthia Tifft , Michael Wangler , Sharayu Jangam , Oguz Kanca , Jonathan Andrews , Christinev Malicdan , Biancae Russell , Hugoj Bellen , Los Angeles , Stanleyf Nelson , Emily Henderson , Diseases Program , Baylor College Of Medicine , University Of California , Diseases Network , National Institutes Of Health , Researchers At Baylor College Of Medicine , Dan Duncan Neurological Research Institute , Baylor College , Undiagnosed Diseases Network , Dan Duncan Neurological Research , Jung Wan Mok , Lee Kai Wang ,

Rare disease, fatal diagnosis doesn't stop woman from bucket list life

Jessie Jackson won t benefit from a new treatment, but the North Carolina woman s participation in a study on her rare condition will help others. ....

Winston Salem , North Carolina , United States , North Wilkesboro , Jessie Jackson , James Mulcahy Jr , Forrest Gump , Patrick Oehler , Clint Zimmerman , Cynthia Tifft , National Institutes Of Health , Diseases Association , National Institutes , National Tay Sachs , James Mulcahy , Disney World , Nitro Harley , Sio Gene Therapies , Allied Diseases ,

Sio Gene Therapies reports positive interim data for gene therapy trial

Sio Gene Therapies, a clinical-stage company focused on developing gene therapies for neurodegenerative diseases, presented positive interim data from its Phase I/II study of AXO-AAV-GM1, an adeno-associated viral vector-based gene therapy candidate for GM1 gangliosidosis that was licensed from UMass Chan. ....

Umass Chan , Douglas Martin , Heather Gray Edwards , Cynthia Tifft , Miguel Sena Esteves , Gavin Corcoran , National Human Genome Research Institute , Auburn University College Of Veterinary Medicine , Umass Chan Medical School , Sio Gene Therapies , Chan Medical School , Auburn University College , Veterinary Medicine , European Society , Cell Therapy Virtual Congress , Sio Gene , Floor Mobility ,