1. Diversity oriented biosynthesis via accelerated evolution of modular gene clusters. Issue 1 (December 2017) Authors: Wlodek, Aleksandra; Kendrew, Steve; Coates, Nigel; Hold, Adam; Pogwizd, Joanna; Rudder, Steven; Sheehan, Lesley; Higginbotham, Sarah; Stanley-Smith, Anna; Warneck, Tony; Nur-E-Alam, Mohammad; Radzom, Markus; Martin, Christine; Overvoorde, Lois; Samborskyy, Markiyan; Alt, Silke; Heine, Daniel; Car... Journal: Nature communications Issue: Volume 8:Issue 1(2017) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier. Issue 1 (16th November 2018) Authors: 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 Journal: British journal of clinical pharmacology Issue: Volume 85:Issue 1(2019) Page Start: 181 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. SMN protein is required throughout life to prevent spinal muscular atrophy disease progression. Issue 1 (9th August 2021) Authors: 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 Journal: Human molecular genetics Issue: Volume 31:Issue 1(2022) Page Start: 82 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers. Issue 1 (December 2017) Authors: Sivaramakrishnan, Manaswini; McCarthy, Kathleen; Campagne, Sébastien; Huber, Sylwia; Meier, Sonja; Augustin, Angélique; Heckel, Tobias; Meistermann, Hélène; Hug, Melanie; Birrer, Pascale; Moursy, Ahmed; Khawaja, Sarah; Schmucki, Roland; Berntenis, Nikos; Giroud, Nicolas; Golling, Sabrina; Tzouros... Journal: Nature communications Issue: Volume 8:Issue 1(2017) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset. Issue 9 (17th November 2020) Authors: 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 Journal: Human molecular genetics Issue: Volume 30:Issue 9(2021) Page Start: 843 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Issue 6 (29th November 2018) Authors: 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 Journal: Pharmacology research & perspectives Issue: Volume 6:Issue 6(2018) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies. (June 2017) Authors: Marquet, Anne; Sturm, Stefan; Kletzl, Heidemarie; Günther, Andreas; Czech, Christian; Poirier, Agnes; Seabrook, Timothy; Müller, Lutz; Kinch, Russell; Ratni, Hasane; Gorni, Ksenija; Metzger, Friedrich; Khwaja, Omar Journal: European journal of paediatric neurology Issue: Volume 21(2017)Supplement 1 Page Start: e226 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗