Friedrich Metzger

<i>SMN2</i> splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Nikolai A. Naryshkin, Marla Weetall, Amal Dakka et al. · Science · 2014 · 526 citations

Life Sciences, Molecular Biology, Neuroscience +17

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( <i>SMN2</i> ) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Hasane Ratni, Martin Ebeling, John D. Baird et al. · Journal of Medicinal Chemistry · 2018 · 520 citations · Full text

Medicine, Neurology, Pharmacology +14

Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

Manaswini Sivaramakrishnan, Kathleen McCarthy, Sébastien Campagne et al. · Nature Communications · 2017 · 221 citations · Full text

Life Sciences, Molecular Biology, Neuroscience +12

Amyloid-β Peptide Induces Mitochondrial Dysfunction by Inhibition of Preprotein Maturation

Dirk Mossmann, F.‐Nora Vögtle, Aslı Aras Taşkin et al. · Cell Metabolism · 2014 · 210 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +14

Therapeutic actions of insulin-like growth factor I on APP/PS2 mice with severe brain amyloidosis

Eva Carro, José Luís Trejo, Anthony N. Gerber et al. · Neurobiology of Aging · 2005 · 172 citations

Life Sciences, Neurobiology, Neuroscience +17