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Publications relevant to Phoenix Nest programs; authored, funded, or in collaboration with employees of Phoenix Nest and or Jonah’s Just Begun-Foundation to Cure Sanfilippo.

Cheng KW, Wang F, Lopez GA, Singamsetty S, Wood J, Dickson PI, Chou TF. Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells. Biochem J. 2021 Jun 25;478(12):2309-2319. doi: 10.1042/BCJ20210015. PMID: 34032266.


Wang F, et al. (2020). Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice.  Mol Pharm. 2021 Jan 4;18(1):214-227. doi: 10.1021/acs.molpharmaceut.0c00831. Epub 2020 Dec 15. PMID: 33320673


Heon-Roberts R, Nguyen ALA, Pshezhetsky AV. Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease.  J Clin Med. 2020 Jan 27;9(2):344. doi: 10.3390/jcm9020344. PMID: 32012694


Martins C, et al. (2019). Molecular characterization of a large group of Mucopolysaccharidosis type IIIC patients reveals the evolutionary history of the disease.  Hum Mutat. 2019 Aug;40(8):1084-1100. doi: 10.1002/humu.23752. Epub 2019 Jun 22. PMID: 31228227


Ekins S, Wood J.  Incentives for Starting Small Companies Focused on Rare and Neglected Diseases. 2016 Apr;33(4):809-15. doi: 10.1007/s11095-015-1841-9. Epub 2015 Dec 14. PMID: 26666772


Carla Martins, et al. (2015) Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model.  Brain 2015 Feb;138(Pt 2):336-55. doi: 10.1093/brain/awu355. Epub 2015 Jan 6. PMID: 25567323


Wood J, Sames L, Moore A, Ekins S. Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery.  Drug Discov Today 2013 Nov;18(21-22):1043-51. doi: 10.1016/j.drudis.2013.08.006. Epub 2013 Aug 19. PMID: 23968993

Presentations & Press

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