P142: Efficacy, safety and tolerability of chenodeoxycholic acid in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized, placebo-controlled phase 3 study

Autor: Andrea DeBarber, Yaz Kisanuki, Paulo Nobrega, Ryan Himes, Suman Jayadev, John Bernat, Vikram Prakash, James Gibson, Austin Larson, Paulo Sgobbi, Edward Murphy, Brian Fedor, Cheryl Wong Po Foo, Rana Dutta, Michael Imperiale, Jolan Terner-Rosenthal, Will Garner, Pamela Vig, P. Barton Duell, Sarah Perez, Ritesh Ramdhani, Jonas Saute

Publikováno v: Genetics in Medicine Open, Vol 2, Iss , Pp 101039- (2024)

Externí odkaz: https://doaj.org/article/42357f5775fb45ad9cbb1f099d5a0e41

2022 Acknowledgment of mSphere Ad Hoc Reviewers

Autor: Michael Imperiale

Publikováno v: mSphere. 7

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::2a7c2da879f8aa1837b9d5da41d9b0cb
https://doi.org/10.1128/msphere.00543-22

A phase 2, randomized, double-blind, placebo-controlled, multicentre study to evaluate the efficacy and safety of pentosan polysulfate sodium in treating subjects with mucopolysaccharidosis type VI: Study update

Autor: Roberto Giugliani, Paula Frassinetti Vasconcelos de Medeiros, Divya Navuru, Curtis Gravance, Ravi Krishnan, Michael Eldon, Michael Imperiale

Publikováno v: Molecular Genetics and Metabolism. 138:107119

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::89676e76f3bac79fc666161a3dd9e312
https://doi.org/10.1016/j.ymgme.2022.107119

A double-blind placebo-controlled phase 2 study to evaluate the safety and tolerability of pentosan polysulfate sodium in subjects with mucopolysaccharidosis type VI (MPS VI)

Autor: Roberto Giugliani, Paula Frassinetti Vasconcelos de Medeiros, Michael Imperiale, Ravi Krishnan, Michael Eldon, Sharon Charles

Publikováno v: Molecular Genetics and Metabolism. 135:S47

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::889404c8f1ab68492331d8e5cc7290a5
https://doi.org/10.1016/j.ymgme.2021.11.111

Acknowledgment of Ad Hoc Reviewers

Autor: Michael Imperiale

Publikováno v: mSphere, Vol 1, Iss 6 (2016)
mSphere
mSphere, Vol 1, Iss 6, p e00358-16 (2016)
mSphere, Vol 3, Iss 6, p e00648-18 (2018)
mSphere, Vol 3, Iss 6 (2018)
mSphere, Vol 2, Iss 6, p e00543-17 (2017)
mSphere, Vol 2, Iss 6 (2017)
mSphere, Vol 5, Iss 6 (2020)
mSphere, Vol 4, Iss 6, p e00873-19 (2019)
mSphere, Vol 4, Iss 6 (2019)

As we reach the end of our first full year of publishing cutting-edge research in the microbial sciences, we would like to offer our sincere thanks to the many individuals who have acted as referees of the manuscripts that we have handled since our b

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::259137b58da0cde1fd945fd085847d05
https://journals.asm.org/doi/10.1128/mSphere.00358-16