Addressing the needs of nano-rare patients: the n-Lorem experience.
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| Title: | Addressing the needs of nano-rare patients: the n-Lorem experience. |
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| Authors: | Crooke ST; n-Lorem Foundation, Carlsbad, CA 92101, United States., Glass S; n-Lorem Foundation, Carlsbad, CA 92101, United States., Gleeson JG; Department of Neurosciences, University of California, San Diego, CA 92093, United States.; Rady Children's Institute for Genomic Medicine, San Diego, CA 92123. United States., Mignon L; n-Lorem Foundation, Carlsbad, CA 92101, United States., Skourti-Stathaki K; n-Lorem Foundation, Carlsbad, CA 92101, United States., Douville J; n-Lorem Foundation, Carlsbad, CA 92101, United States., Knutsen M; n-Lorem Foundation, Carlsbad, CA 92101, United States., Pu H; n-Lorem Foundation, Carlsbad, CA 92101, United States., Bain JM; Department of Neurology, Division of Child Neurology, Columbia University Irving Medical Center, New York, NY 10032, United States., Berry-Kravis E; Department of Pediatrics, Neurological Sciences and Anatomy and Cell Biology, Rush University Medical Center, Chicago, IL 60612, United States., Shneider NA; Center for Motor Neuron Biology and Disease, Columbia University Irving Medical Center, New York, NY 10032, United States.; Department of Neurology, Eleanor and Lou Gehrig ALS Center, Columbia University Irving Medical Center, New York, NY 10032, United States., Kim-McManus O; UCSD Rady Children's Institute for Genomic Medicine, San Diego, CA 92123, United States., Eichler FS; Department of Neurology, Harvard Medical School, Boston, MA 02115, United States.; Department of Neurology, Massachusetts General Research Institute, Boston, MA 02114, United States., Chung WK; Department Pediatrics at Boston Children's Hospital, Boston, MA 02115, United States.; Harvard Medical School, Boston, MA 02115, United States., Nagy A; Department of Neurology, Harvard Medical School, Boston, MA 02115, United States.; Department of Neurology, Massachusetts General Research Institute, Boston, MA 02114, United States., Kaufmann H; Department of Neurology, New York University School of Medicine, New York, NY 10017, United States., Gonzalez-Duarte A; Department of Neurology, Dysautonomia Center, NYU Langone Health, NYU Grossman school of Medicine, New York, NY 10016, United States., Oskarsson B; Mayo Clinic, Jacksonville, FL 55905, United States., McCourt EA; Department of Ophthalmology, University of Colorado School of Medicine, Children's Hospital of Colorado, Anschutz Medical Campus, Aurora, CO 80045, United States., Leung N; Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, MN 55905, United States.; Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN 55905, United States. |
| Source: | Nucleic acids research [Nucleic Acids Res] 2026 May 20; Vol. 54 (10). |
| Publication Type: | Journal Article; Review |
| Journal Info: | Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1362-4962 |
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| DOI: | 10.1093/nar/gkag504 |
