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Scientists reverse the effects of lysosomal storage disorders in patient cells and mice

Scientists at the Icahn School of Medicine at Mount Sinai in New York, the National Center for Advancing Translational Sciences (NCATS), and elsewhere have reversed the effects of several life-threatening inherited neurodegenerative diseases called lysosomal storage disorders (LSDs) in patient cells and mice.
New York United States Yiannis Ioannou Juan Marugan Emily Henderson National Center

Sangamo Therapeutics, Inc.: Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data in Fabry Disease Clinical Study Showing Continued Tolerability and Sustained Elevated a-gal A Enzyme Activity in Five Longest Treated Patients

Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across three dose cohorts in the six treated patients The five longest treated patients exhibited elevated a-Gal A activity
Nathalie Dubois Stringfellow Louise Wilkie Jaya Ganesh Development Officer Division Of Medical Genetics Drug Administration

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Mount Sinai and Regeneron Genetics Center launch a new human genome sequencing research project

Mount Sinai Health System and the Icahn School of Medicine at Mount Sinai have launched a new human genome sequencing research project called the Mount Sinai Million Health Discoveries Program with the Regeneron Genetics Center (RGC), part of the industry-leading, New York-based biotechnology company Regeneron.
Dara Meyer Girishn Nadkarni Rachelle Weisman Emily Henderson Ericj Nestler Aris Baras

Mount Sinai receives $500,000 SU2C grant to identify therapeutic combinations for KRAS-mutant lung cancers

Mount Sinai is leading a team of lung cancer researchers that has been awarded a three-year, $500,000 grant from Stand Up To Cancer to explore therapeutic approaches to lung tumors with mutations in the KRAS gene, an undertaking that could have a significant impact for a large population of cancer patients who currently lack effective treatment options.
Fredr Hirsch Benjamind Hopkins Joe Lowe Davidh Cogan Anne Murnick Cogan Emily Henderson

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