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 · 517 citations

Genetics, Molecular Biology, Therapeutics +21

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 · 492 citations · Full text

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 · 219 citations · Full text

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

Dirk Mossmann, F.‐Nora Vögtle, Aslı Aras Taşkin et al. · Cell Metabolism · 2014 · 206 citations · Full text

Alzheimer's Disease, Mitochondrial Function, Biochemistry +8

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 · 171 citations

Alzheimer's Disease, Therapeutic Actions, Diabetes +9