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1.
SMN protein is required throughout life to prevent spinal muscular atrophy disease progression.
Hum Mol Genet
; 31(1): 82-96, 2021 12 17.
Artículo
en Inglés
| MEDLINE | ID: mdl-34368854
2.
Structural basis of a small molecule targeting RNA for a specific splicing correction.
Nat Chem Biol
; 15(12): 1191-1198, 2019 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-31636429
3.
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.
Chimia (Aarau)
; 75(7): 614-619, 2021 Aug 25.
Artículo
en Inglés
| MEDLINE | ID: mdl-34523402
4.
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
Br J Clin Pharmacol
; 85(1): 181-193, 2019 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-30302786
5.
Rewriting the (tran)script: Application to spinal muscular atrophy.
Prog Med Chem
; 58: 119-156, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-30879473
6.
Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Hum Mol Genet
; 25(5): 964-75, 2016 Mar 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-26758873
7.
Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
Hum Mol Genet
; 30(9): 843, 2021 May 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-33201218
8.
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Hum Mol Genet
; 25(10): 1885-1899, 2016 05 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-26931466
9.
Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines.
ACS Med Chem Lett
; 12(6): 874-877, 2021 Jun 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-34141064
10.
Discovery of RO7185876, a Highly Potent γ-Secretase Modulator (GSM) as a Potential Treatment for Alzheimer's Disease.
ACS Med Chem Lett
; 11(6): 1257-1268, 2020 Jun 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-32551009
11.
Discovery of Balovaptan, a Vasopressin 1a Receptor Antagonist for the Treatment of Autism Spectrum Disorder.
J Med Chem
; 63(4): 1511-1525, 2020 02 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-31951127
12.
Me-talnetant and osanetant interact within overlapping but not identical binding pockets in the human tachykinin neurokinin 3 receptor transmembrane domains.
Mol Pharmacol
; 73(6): 1736-50, 2008 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-18308898
13.
Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
Pharmacol Res Perspect
; 6(6): e00447, 2018 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-30519476
14.
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).
J Med Chem
; 61(15): 6501-6517, 2018 Aug 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-30044619
15.
Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy.
J Med Chem
; 60(10): 4444-4457, 2017 05 25.
Artículo
en Inglés
| MEDLINE | ID: mdl-28441483
16.
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers.
Nat Commun
; 8(1): 1476, 2017 11 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-29133793
17.
Diversity oriented biosynthesis via accelerated evolution of modular gene clusters.
Nat Commun
; 8(1): 1206, 2017 10 31.
Artículo
en Inglés
| MEDLINE | ID: mdl-29089518
18.
Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
J Med Chem
; 59(13): 6086-100, 2016 07 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-27299419
19.
Discovery of highly selective brain-penetrant vasopressin 1a antagonists for the potential treatment of autism via a chemogenomic and scaffold hopping approach.
J Med Chem
; 58(5): 2275-89, 2015 Mar 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-25654260
20.
Mapping the binding pocket of a novel, high-affinity, slow dissociating tachykinin NK3 receptor antagonist: biochemical and electrophysiological characterization.
Neuropharmacology
; 86: 259-72, 2014 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-25107588