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Friedreich's Ataxia-Health Index: Development and Validation of a Novel Disease-Specific Patient-Reported Outcome Measure.
Seabury J, Rosero S, Varma A, Weinstein J, Engebrecht C, Dilek N, Heatwole J, Alexandrou D, Cohen B, Larkindale J, Lynch DR, Park C, Subramony SH, Wagner E, Walther S, Wells M, Zizzi C, Heatwole C. Seabury J, et al. Among authors: larkindale j. Neurol Clin Pract. 2023 Oct;13(5):e200180. doi: 10.1212/CPJ.0000000000200180. Epub 2023 Aug 28. Neurol Clin Pract. 2023. PMID: 37646046
Patient-Reported Impact of Symptoms in Friedreich Ataxia.
Seabury J, Alexandrou D, Dilek N, Cohen B, Heatwole J, Larkindale J, Lynch DR, Park C, Rosero S, Subramony SH, Varma A, Wagner E, Walther S, Weinstein J, Wells M, Zizzi C, Heatwole C. Seabury J, et al. Among authors: larkindale j. Neurology. 2023 Feb 21;100(8):e808-e821. doi: 10.1212/WNL.0000000000201598. Epub 2022 Nov 28. Neurology. 2023. PMID: 36443012 Free PMC article.
Developing a Natural History Model for Duchenne Muscular Dystrophy.
Broomfield J, Hill M, Chandler F, Crowther MJ, Godfrey J, Guglieri M, Hastie J, Larkindale J, Mumby-Croft J, Reuben E, Woodcock F, Abrams KR; Project HERCULES, the Cooperative International Neuromuscular Research Group investigators, Duchenne Regulatory Science Consortium members. Broomfield J, et al. Among authors: larkindale j. Pharmacoecon Open. 2024 Jan;8(1):79-89. doi: 10.1007/s41669-023-00450-x. Epub 2023 Nov 29. Pharmacoecon Open. 2024. PMID: 38019449 Free PMC article.
The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies.
Beaverson KL, Julkowska D, Letinturier MCV, Aartsma-Rus A, Austin J, Bueren J, Frost S, Hamamura M, Larkindale J, LaRosa G, Magenheim R, Merico A, Gerdina Pasmooij AM, Pirard V, Ekow Thomford N, Wada M, Wong-Rieger D, Hartman AL. Beaverson KL, et al. Among authors: larkindale j. Ther Adv Rare Dis. 2023 Jul 29;4:26330040231188979. doi: 10.1177/26330040231188979. eCollection 2023 Jan-Dec. Ther Adv Rare Dis. 2023. PMID: 37529076 Free PMC article.
Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy.
Lingineni K, Aggarwal V, Morales JF, Conrado DJ, Corey D, Vong C, Burton J, Larkindale J, Romero K, Schmidt S, Kim S; Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members. Lingineni K, et al. Among authors: larkindale j. CPT Pharmacometrics Syst Pharmacol. 2022 Mar;11(3):318-332. doi: 10.1002/psp4.12753. Epub 2022 Jan 3. CPT Pharmacometrics Syst Pharmacol. 2022. PMID: 34877803 Free PMC article.
The Responsiveness of Gait and Balance Outcomes to Disease Progression in Friedreich Ataxia.
Milne SC, Kim SH, Murphy A, Larkindale J, Farmer J, Malapira R, Danoudis M, Shaw J, Ramakrishnan T, Rasouli F, Yiu EM, Georgiou-Karistianis N, Tai G, Zesiewicz T, Delatycki MB, Corben LA. Milne SC, et al. Among authors: larkindale j. Cerebellum. 2022 Dec;21(6):963-975. doi: 10.1007/s12311-021-01348-2. Epub 2021 Dec 2. Cerebellum. 2022. PMID: 34855135
Effective Data Sharing as a Conduit for Advancing Medical Product Development.
Karpen SR, White JK, Mullin AP, O'Doherty I, Hudson LD, Romero K, Sivakumaran S, Stephenson D, Turner EC, Larkindale J. Karpen SR, et al. Among authors: larkindale j. Ther Innov Regul Sci. 2021 May;55(3):591-600. doi: 10.1007/s43441-020-00255-8. Epub 2021 Jan 4. Ther Innov Regul Sci. 2021. PMID: 33398663 Free PMC article. Review.
29 results