Below are the most recent publications written about "Rare Diseases" by people in Profiles.
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Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, B?nnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M. Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther. 2022 07 06; 30(7):2416-2428.
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Bettio C, Salsi V, Orsini M, Calanchi E, Magnotta L, Gagliardelli L, Kinoshita J, Bergamaschi S, Tupler R. The Italian National Registry for FSHD: an enhanced data integration and an analytics framework towards Smart Health Care and Precision Medicine for a rare disease. Orphanet J Rare Dis. 2021 11 04; 16(1):470.
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Glenn DA, Hogan SL. Big Data and Glomerular Disease: Uncovering Common Outcomes of Rare Disease. J Am Soc Nephrol. 2021 09; 32(9):2106-2108.
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Pasquini TLS, Goff SL, Whitehill JM. Navigating the U.S. health insurance landscape for children with rare diseases: a qualitative study of parents' experiences. Orphanet J Rare Dis. 2021 07 15; 16(1):313.
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Seckeler MD, Moedano L, Mustacich D, Kalb BT, Saranathan M, Galons JP, Witte MH. Non-contrast MR lymphography of rare lymphatic abnormalities. Lymphology. 2020; 53(3):136-140.
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Gruntman AM, Flotte TR. The rapidly evolving state of gene therapy. FASEB J. 2018 04; 32(4):1733-1740.
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Breneman JC, Donaldson SS, Constine L, Merchant T, Marcus K, Paulino AC, Followill D, Mahajan A, Laack N, Esiashvili N, Haas-Kogan D, Laurie F, Olch A, Ulin K, Hodgson D, Yock TI, Terezakis S, Krasin M, Panoff J, Chuba P, Hua CH, Hess CB, Houghton PJ, Wolden S, Buchsbaum J, Fitzgerald TJ, Kalapurakal JA. The Children's Oncology Group Radiation Oncology Discipline: 15?Years of Contributions to the Treatment of Childhood Cancer. Int J Radiat Oncol Biol Phys. 2018 07 15; 101(4):860-874.
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Liao H, Khan A, Miron PM, Cornejo KM. Mammary Analogue Secretory Carcinoma of the Thyroid Mimicking Locally Advanced Papillary Thyroid Carcinoma: A Rare Case Report. Int J Surg Pathol. 2018 Aug; 26(5):459-463.
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Marecki H, Simons JP, Schanzer A, Arous EJ, Robichaud D, Messina LM. Repeat presentation for middle aortic syndrome 18?years after original repair. J Vasc Surg. 2017 12; 66(6):1876-1877.
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Simon DA, Smith AL, Holzhauer JR. Case Report of an Ectopic Molar Pregnancy in the Presence of an Intrauterine Device. WMJ. 2017 Nov; 116(4):215-217.