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1.
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Proc Natl Acad Sci U S A
; 111(41): 14870-5, 2014 Oct 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-25267636
2.
Trapping Poly(ADP-Ribose) Polymerase.
J Pharmacol Exp Ther
; 353(3): 446-57, 2015 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-25758918
3.
Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism.
J Pharmacol Exp Ther
; 353(1): 132-49, 2015 Apr.
Artículo
en Inglés
| MEDLINE | ID: mdl-25650377
4.
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
Drug Deliv Transl Res
; 10(2): 425-439, 2020 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-31942701
5.
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B.
Mol Ther Methods Clin Dev
; 14: 56-63, 2019 Sep 13.
Artículo
en Inglés
| MEDLINE | ID: mdl-31309128
6.
BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.
PLoS One
; 14(1): e0207836, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-30657762
7.
Utilizing ExAC to assess the hidden contribution of variants of unknown significance to Sanfilippo Type B incidence.
PLoS One
; 13(7): e0200008, 2018.
Artículo
en Inglés
| MEDLINE | ID: mdl-29979746
8.
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
Mol Ther Methods Clin Dev
; 6: 43-53, 2017 Sep 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-28664165
9.
Discovery and Characterization of (8S,9R)-5-Fluoro-8-(4-fluorophenyl)-9-(1-methyl-1H-1,2,4-triazol-5-yl)-2,7,8,9-tetrahydro-3H-pyrido[4,3,2-de]phthalazin-3-one (BMN 673, Talazoparib), a Novel, Highly Potent, and Orally Efficacious Poly(ADP-ribose) Polymerase-1/2 Inhibitor, as an Anticancer Agent.
J Med Chem
; 59(1): 335-57, 2016 Jan 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-26652717
10.
Structural basis for the inhibition of poly(ADP-ribose) polymerases 1 and 2 by BMN 673, a potent inhibitor derived from dihydropyridophthalazinone.
Acta Crystallogr F Struct Biol Commun
; 70(Pt 9): 1143-9, 2014 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-25195882
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