Bibliography
- RARE List™. Global Genes, Allies in Rare Diseases [Internet]. 2015. [ cited 2015 Nov 11]. Available from: https://globalgenes.org/rarelist/.
- For the purposes of this article, drug repurposing will be defined as finding a new disease indication for an already approved drug. Drug repositioning will be defined as finding a disease indication for a drug-like compound that has been found safe in humans through clinical testing, though it that failed for efficacy or other reasons during development for the initial disease indication(s).
- FDA Approval for Thalidomide. National Cancer Institute [Internet]. 2013. [ cited 2015 Nov 11]. Available from: http://www.cancer.gov/about-cancer/treatment/drugs/fda-thalidomide.
- Barnett C, Machado R. Sildenafil in the treatment of pulmonary hypertension. Vasc Health Risk Manag. 2006;2(4):411–422.
- FDA approves new multiple sclerosis treatment: Tecfidera [Internet]. U.S. Department of Health and Human Services; 2013 [ cited 2015 Nov 11]. Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm345528.htm.
- Kumar S, Witzig T, Rajkumar S. Thalidomide: Current Role in the Treatment of Non-Plasma Cell Malignancies. J Clin Oncol. 2004;22(12):2477–2488.
- Teachey DT, Greiner R, Seif A, et.al. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. Br J Haematol. 2009;145(1):101–106.
- Funding Partner Report March 2015. Cures Within Reach [Internet]. 2015. [ cited 2015 Nov 11]. Available from: http://cureswithinreach.org/images/ProjectReports/LDOGTeachey_Report_March2015.pdf.
- Anderson SL1, Liu B, Qiu J, et al. Nutraceutical-mediated restoration of wild-type levels of IKBKAP-encoded IKAP protein in familial dysautonomia-derived cells. Mol Nutr Food Res. 2012;56(4):570–579.
- FD NOW website. Treatment breakthroughs in FD research from the Fordham Laboratory for familial dysautonomia research [Internet]. 2015. [ cited 2015 Nov 11]. Available from: http://www.fdnow.org/research/treatment-breakthroughs.
- A Phase I Study of Mebendazole for the Treatment of Pediatric Gliomas, Clinicaltrials.gov [Internet]. 2015. [ cited 2015 Nov 11. Available from: https://clinicaltrials.gov/ct2/show/NCT01837862?term=mebendazole+pediatric+glioma&rank=1.
- Suitability of Nitisinone in Alkaptonuria 2 (SONIA 2), Clinicaltrials.gov [Internet]. 2015. [ cited 2015 Nov 11]. Available from: https://clinicaltrials.gov/ct2/show/NCT01916382?term=nitisinone&rank=5.
- Ghosh A, Corbett G, Pahan K, et.al. Gemfibrozil and Fenofibrate, ood and Drug Administration-approved Lipid-lowering Drugs, Up-regulate Tripeptidyl- Tripeptidyl-peptidase 1 in Brain Cells via Peroxisome Proliferator-activated Receptor α. J Biol Chem. 2012;287(46):38922–38935.
- Kast RE, Karpel-Massler G, Halatsch ME. CUSP9* treatment protocol for recurrent glioblastoma: aprepitant, artesunate, auranofin, captopril, celecoxib, disulfiram, itraconazole, ritonavir, sertraline augmenting continuous low dose temozolomide. Oncotarget. 2014;5(18):8052–8082.
- H.R.6 - 21st Century Cures Act. Congress.gov, 2015. [ cited 2015 Nov 11]. Available from: https://www.congress.gov/bill/114th-congress/house-bill/6.