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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2013 4
2014 3
2015 4
2016 4
2017 3
2018 2
2019 7
2020 4
2021 8
2022 9
2023 1
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44 results
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Page 1
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Mendell JR, et al. Among authors: alfano ln. JAMA Neurol. 2020 Sep 1;77(9):1122-1131. doi: 10.1001/jamaneurol.2020.1484. JAMA Neurol. 2020. PMID: 32539076 Free PMC article. Clinical Trial.
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.
Waldrop MA, Karingada C, Storey MA, Powers B, Iammarino MA, Miller NF, Alfano LN, Noritz G, Rossman I, Ginsberg M, Mosher KA, Broomall E, Goldstein J, Bass N, Lowes LP, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Among authors: alfano ln. Pediatrics. 2020 Sep;146(3):e20200729. doi: 10.1542/peds.2020-0729. Pediatrics. 2020. PMID: 32843442
Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy.
Korb M, Peck A, Alfano LN, Berger KI, James MK, Ghoshal N, Healzer E, Henchcliffe C, Khan S, Mammen PPA, Patel S, Pfeffer G, Ralston SH, Roy B, Seeley WW, Swenson A, Mozaffar T, Weihl C, Kimonis V; VCP Standards of Care Working Group. Korb M, et al. Among authors: alfano ln. Orphanet J Rare Dis. 2022 Jan 29;17(1):23. doi: 10.1186/s13023-022-02172-5. Orphanet J Rare Dis. 2022. PMID: 35093159 Free PMC article. Review.
Genotype-phenotype correlations in valosin-containing protein disease: a retrospective muticentre study.
Schiava M, Ikenaga C, Villar-Quiles RN, Caballero-Ávila M, Topf A, Nishino I, Kimonis V, Udd B, Schoser B, Zanoteli E, Souza PVS, Tasca G, Lloyd T, Lopez-de Munain A, Paradas C, Pegoraro E, Nadaj-Pakleza A, De Bleecker J, Badrising U, Alonso-Jiménez A, Kostera-Pruszczyk A, Miralles F, Shin JH, Bevilacqua JA, Olivé M, Vorgerd M, Kley R, Brady S, Williams T, Domínguez-González C, Papadimas GK, Warman-Chardon J, Claeys KG, de Visser M, Muelas N, LaForet P, Malfatti E, Alfano LN, Nair SS, Manousakis G, Kushlaf HA, Harms MB, Nance C, Ramos-Fransi A, Rodolico C, Hewamadduma C, Cetin H, García-García J, Pál E, Farrugia ME, Lamont PJ, Quinn C, Nedkova-Hristova V, Peric S, Luo S, Oldfors A, Taylor K, Ralston S, Stojkovic T, Weihl C, Diaz-Manera J; VCP International Study Group; VCP International Study Group. Schiava M, et al. Among authors: alfano ln. J Neurol Neurosurg Psychiatry. 2022 Jul 27:jnnp-2022-328921. doi: 10.1136/jnnp-2022-328921. Online ahead of print. J Neurol Neurosurg Psychiatry. 2022. PMID: 35896379
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.
Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Lowes LP, et al. Among authors: alfano ln. Pediatr Neurol. 2019 Sep;98:39-45. doi: 10.1016/j.pediatrneurol.2019.05.005. Epub 2019 May 13. Pediatr Neurol. 2019. PMID: 31277975 Free article. Clinical Trial.
Comparison of strength testing modalities in dysferlinopathy.
Reash NF, James MK, Alfano LN, Mayhew AG, Jacobs M, Iammarino MA, Holsten S, Sakamoto C, Tateishi T, Yajima H, Duong T, de Wolf B, Gee R, Bharucha-Goebel DX, Bravver E, Mori-Yoshimura M, Bushby K, Rufibach LE, Straub V, Lowes LP; Jain COS Consortium. Reash NF, et al. Among authors: alfano ln. Muscle Nerve. 2022 Aug;66(2):159-166. doi: 10.1002/mus.27570. Epub 2022 May 20. Muscle Nerve. 2022. PMID: 35506767
44 results