Clinical trials involving treatments for rare disease such as pseudoxanthoma elasticum [PXE] are difficult to sponsor and maintain due to the lack of foreseeable gain on the part of the treatment developer. However, PXE International is currently working with multiple organizations to help develop a clinical trial on PXE.
Check on the progress: Clinical Trial of Magnesium Supplementation as a Treatment for PXE
The benefits of participant ownership and sharing of clinical trial data appear to outweigh the risks. Read more in Power to the People: Participant Ownership of Clinical Trial Data, by Sharon and Patrick Terry, published February 9, 2011 in Science Translational Medicine.
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Uitto J, Bercovitch L, Terry SF, Terry PF. Pseudoxanthoma elasticum: Progress in diagnostics and research towards treatment : Summary of the 2010 PXE International Research Meeting. Am J Med Genet A. 2011 Jul;155(7):1517-26. Free article in PubMed. PMID: 21671388
Lee TK, Forooghian F, Cukras C, Wong WT, Chew EY, Meyerle CB. Complementary angiographic and autofluorescence findings in pseudoxanthoma elasticum. Int Ophthalmol. 2010 Feb;30(1):77-9. Free article in PubMed. PMID: 18854947
Bhatnagar P, Freund KB, Spaide RF, Klancnik JM Jr, Cooney MJ, Ho I, Fine HF, Yannuzzi LA. Intravitreal bevacizumab for the management of choroidal neovascularization in pseudoxanthoma elasticum. Retina. 2007 Sep;27(7):897-902. PMID: 17891014