Furlong P - Search Results

1

Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints.

Kakkis ED, O'Donovan M, Cox G, Hayes M, Goodsaid F, Tandon PK, Furlong P, Boynton S, Bozic M, Orfali M, Thornton M. Kakkis ED, et al. Among authors: furlong p. Orphanet J Rare Dis. 2015 Feb 10;10:16. doi: 10.1186/s13023-014-0195-4. Orphanet J Rare Dis. 2015. PMID: 25757705 Free PMC article. Review.

2

Flow cytometric assessment of monocyte activation markers and circulating endothelial cells in patients with localized or metastatic breast cancer.

Goodale D, Phay C, Brown W, Gray-Statchuk L, Furlong P, Lock M, Chin-Yee I, Keeney M, Allan AL. Goodale D, et al. Among authors: furlong p. Cytometry B Clin Cytom. 2009 Mar;76(2):107-17. doi: 10.1002/cyto.b.20449. Cytometry B Clin Cytom. 2009. PMID: 18727054 Free article.

3

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.

Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT. Heslop E, et al. Orphanet J Rare Dis. 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1. Orphanet J Rare Dis. 2015. PMID: 25902795 Free PMC article. Review.

4

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.

Furlong P, Bridges JF, Charnas L, Fallon JR, Fischer R, Flanigan KM, Franson TR, Gulati N, McDonald C, Peay H, Sweeney HL. Furlong P, et al. Orphanet J Rare Dis. 2015 Jun 24;10:82. doi: 10.1186/s13023-015-0281-2. Orphanet J Rare Dis. 2015. PMID: 26104810 Free PMC article.

5

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.

Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Among authors: furlong p. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.

6

Helpful Lessons and Cautionary Tales: How Should COVID-19 Drug Development and Access Inform Approaches to Non-Pandemic Diseases?

Lynch HF, Caplan A, Furlong P, Bateman-House A. Lynch HF, et al. Among authors: furlong p. Am J Bioeth. 2021 Dec;21(12):4-19. doi: 10.1080/15265161.2021.1974975. Epub 2021 Oct 19. Am J Bioeth. 2021. PMID: 34665689

7

Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials.

Furlong P, Dugar A, White M. Furlong P, et al. Res Involv Engagem. 2024 Jan 2;10(1):1. doi: 10.1186/s40900-023-00535-1. Res Involv Engagem. 2024. PMID: 38167117 Free PMC article.

8

A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy.

Crossnohere NL, Fischer R, Vroom E, Furlong P, Bridges JFP. Crossnohere NL, et al. Among authors: furlong p. Patient. 2022 Sep;15(5):577-588. doi: 10.1007/s40271-022-00574-y. Epub 2022 Mar 4. Patient. 2022. PMID: 35243571 Free PMC article.

9

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.

Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Bushby K, et al. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27. Lancet Neurol. 2010. PMID: 19945913 Review.

10

The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009.

Muntoni F; Meeting Steering Committee and TREAT-NMD Network. Muntoni F, et al. Neuromuscul Disord. 2010 May;20(5):355-62. doi: 10.1016/j.nmd.2010.03.005. Epub 2010 Mar 26. Neuromuscul Disord. 2010. PMID: 20347306 No abstract available.

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