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Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
Published in:
Pharmacology Research & Perspectives, 2018, v. 6, n. 6, p. 1, doi. 10.1002/prp2.447
By:
- Poirier, Agnès;
- Weetall, Marla;
- Heinig, Katja;
- Bucheli, Franz;
- Schoenlein, Kerstin;
- Alsenz, Jochem;
- Bassett, Simon;
- Ullah, Mohammed;
- Senn, Claudia;
- Ratni, Hasane;
- Naryshkin, Nikolai;
- Paushkin, Sergey;
- Mueller, Lutz
Publication type:
Article
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
Published in:
British Journal of Clinical Pharmacology, 2019, v. 85, n. 1, p. 181, doi. 10.1111/bcp.13786
By:
- Sturm, Stefan;
- Günther, Andreas;
- Jaber, Birgit;
- Jordan, Paul;
- Al Kotbi, Nada;
- Parkar, Nikhat;
- Cleary, Yumi;
- Frances, Nicolas;
- Bergauer, Tobias;
- Heinig, Katja;
- Kletzl, Heidemarie;
- Marquet, Anne;
- Ratni, Hasane;
- Poirier, Agnès;
- Müller, Lutz;
- Czech, Christian;
- Khwaja, Omar
Publication type:
Article
SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
Published in:
Human Molecular Genetics, 2022, v. 31, n. 1, p. 82, doi. 10.1093/hmg/ddab220
By:
- Zhao, Xin;
- Feng, Zhihua;
- Risher, Nicole;
- Mollin, Anna;
- Sheedy, Josephine;
- Ling, Karen K Y;
- Narasimhan, Jana;
- Dakka, Amal;
- Baird, John D;
- Ratni, Hasane;
- Lutz, Catherine;
- Chen, Karen S;
- Naryshkin, Nikolai A;
- Ko, Chien-Ping;
- Welch, Ellen;
- Metzger, Friedrich;
- Weetall, Marla
Publication type:
Article
Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Published in:
Human Molecular Genetics, 2021, v. 30, n. 9, p. 843, doi. 10.1093/hmg/ddaa247
By:
- Feng, Zhihua;
- Ling, Karen K Y;
- Zhao, Xin;
- Zhou, Chunyi;
- Karp, Gary;
- Welch, Ellen M;
- Naryshkin, Nikolai;
- Ratni, Hasane;
- Chen, Karen S;
- Metzger, Friedrich;
- Paushkin, Sergey;
- Weetall, Marla;
- Ko, Chien-Ping
Publication type:
Article
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Published in:
Human Molecular Genetics, 2016, v. 25, n. 10, p. 1885, doi. 10.1093/hmg/ddw062
By:
- Xin Zhao;
- Zhihua Feng;
- Ling, Karen K. Y.;
- Mollin, Anna;
- Sheedy, Josephine;
- Yeh, Shirley;
- Petruska, Janet;
- Narasimhan, Jana;
- Dakka, Amal;
- Welch, Ellen M.;
- Karp, Gary;
- Chen, Karen S.;
- Metzger, Friedrich;
- Ratni, Hasane;
- Lotti, Francesco;
- Tisdale, Sarah;
- Naryshkin, Nikolai A.;
- Pellizzoni, Livio;
- Paushkin, Sergey;
- Chien-Ping Ko
Publication type:
Article
Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Published in:
Human Molecular Genetics, 2016, v. 25, n. 5, p. 964, doi. 10.1093/hmg/ddv629
By:
- Zhihua Feng;
- Ling, Karen K. Y.;
- Xin Zhao;
- Chunyi Zhou;
- Karp, Gary;
- Welch, Ellen M.;
- Naryshkin, Nikolai;
- Ratni, Hasane;
- Chen, Karen S.;
- Metzger, Friedrich;
- Paushkin, Sergey;
- Weetall, Marla;
- Chien-Ping Ko
Publication type:
Article
Contribution to the Discovery of a Novel Medicine for a Neuromuscular Disease and of other Promising Molecules for the Treatment of Neurodevelopmental and Neurodegenerative Diseases.
Published in:
Chimia, 2021, v. 75, n. 7/8, p. 614, doi. 10.2533/chimia.2021.614
By:
- Ratni, Hasane
Publication type:
Article

Found: 7

Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.

A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.

SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.

Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.

Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.

Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.

Contribution to the Discovery of a Novel Medicine for a Neuromuscular Disease and of other Promising Molecules for the Treatment of Neurodevelopmental and Neurodegenerative Diseases.