Therapeutic Approaches

[listed by category in reverse chronological order]

1. 2015 Regeneron team’s paper on the role of Activin A
Hatsell SJ, Idone V, Wolken DMA, Huang L, Kim HJ, Wang L, Wen X, Nannuru KC, Jimenez J, Xie L, Das N, Makhoul G, Chernomorsky R, D’Ambrosio D, Corpina RA, Schoenherr CJ, Feeley K, Yu PB, Yancopoulos GD, Murphy AJ, Economides AN. ACVR1R206H receptor mutation causes fibrodysplasia ossificans progressiva by imparting responsiveness to activin A. Sci. Transl. Med. 7, 303ra137 (2015). Click here for abstract.

2. 2014 Harvard / Yu lab’s paper on LDN (kinase inhibitor)
Mohedas AH, Wang Y, Sanvitale CE, Canning P, Choi S, Xing X, Bullock AN, Cuny GD, Yu PB. Structure-activity relationship of 3,5-diaryl-2-aminopyridine ALK2 inhibitors reveals unaltered binding affinity for fibrodysplasia ossificans progressiva causing mutants. J Med Chem. 2014 Oct 9;57(19):7900-15.

3. 2014 Carnegie-Melon / Shrivats and Hollinger paper on polymer-based RNAi
Shrivats AR, Hollinger JO. The delivery and evaluation of RNAi therapeutics for heterotopic ossification pathologies. Methods Mol Biol. 2014;1202:149-60. Click here for abstract.

4. 2013 U Penn / Kaplan “From Mysteries to Medicine” review article
Kaplan FS, Pignolo RJ, Shore EM. From mysteries to medicines: drug development for fibrodysplasia ossificans progressive. Expert Opin Orphan Drugs. 2013 Aug;1(8):637-649. Click here for abstract.

5. 2013 Oxford / Bullock lab’s K0288 paper (kinase inhibitor)
Sanvitale CE, Kerr G, Chaikuad A, Ramel MC, Mohedas AH, Reichert S, Wang Y, Triffitt JT, Cuny GD, Yu PB, Hill CS, Bullock AN. A new class of small molecule inhibitor of BMP signaling. PLoS One. 2013 Apr 30;8(4):e62721.

6. 2013 Leiden / ten Dijke lab’s paper on antisense oligonucleotides
Shi S, Cai J, de Gorter DJ, Sanchez-Duffhues G, Kemaladewi DU, Hoogaars WM, Aartsma-Rus A, Hoen PA, ten Dijke P. Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressiva. PLoS One. 2013 Jul 4;8(7):e69096.

7. 2013 Nagoya / Kitoh et al perhexilene maleate study
Kitoh H, Achiwa M, Kaneko H, Mishima K, Matsushita M, Kadono I, Horowitz JD, Sallustio BC, Ohno K, Ishiguro N. Perhexiline maleate in the treatment of fibrodysplasia ossificans progressiva: an open-labeled clinical trial. Orphanet J Rare Dis. 2013 Oct 16;8:163.

8. 2012 Harvard / Lowery and Rosen commentary on RNAi
Lowery JW and Rosen V. Allele-Specific RNA Interference in FOP: Silencing the FOP gene. Gene Therapy (2012) 19, 701–702.

9. 2011 CHOP / Shimono paper on RAR-gamma
Shimono K, Tung WE, Macolino C, Chi AH, Didizian JH, Mundy C, Chandraratna RA, Mishina Y, Enomoto-Iwamoto M, Pacifici M, Iwamoto M. Potent inhibition of heterotopic ossification by nuclear retinoic acid receptor-γ agonists. Nat Med. 2011 Apr;17(4):454-60. Click here for abstract.


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