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A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier
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
Published in British journal of clinical pharmacology (01.01.2019)
Published in British journal of clinical pharmacology (01.01.2019)
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J06 Pivot-HD: a phase 2, randomised, placebo-controlled study to evaluate the safety and efficacy of ptc518 in subjects with Huntington’s disease
Beers, Brian, Bredlau, Amy-Lee, Bhattacharyya, Anu, Kristensen, Allan, Golden, Lee, Klein, Matthew, Peltz, Stuart
Published in Journal of neurology, neurosurgery and psychiatry (01.09.2022)
Published in Journal of neurology, neurosurgery and psychiatry (01.09.2022)
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Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy
Sergott, Robert C., Amorelli, Giulia M., Baranello, Giovanni, Barreau, Emmanuel, Beres, Shannon, Kane, Steven, Mercuri, Eugenio, Orazi, Lorenzo, SantaMaria, Melissa, Tremolada, Gemma, Santarsiero, Diletta, Waskowska, Agnieszka, Yashiro, Shigeko, Denk, Nora, Fürst‐Recktenwald, Sabine, Gerber, Marianne, Gorni, Ksenija, Jaber, Birgit, Jacobsen, Bjoern, Mueller, Lutz, Nave, Stephane, Scalco, Renata S., Marzoli, Stefania B.
Published in Annals of clinical and translational neurology (01.01.2021)
Published in Annals of clinical and translational neurology (01.01.2021)
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