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How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.
- Published in:
- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-015-0281-2
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- Article
MuSK is a BMP co-receptor that shapes BMP responses and calcium signaling in muscle cells.
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- Science Signaling, 2016, v. 9, n. 444, p. 1, doi. 10.1126/scisignal.aaf0890
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- Article
Shaping Membrane Architecture: Agrins In and Out of the Synapse.
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- Journal of Cell Biology, 2001, v. 153, n. 7, p. F39, doi. 10.1083/jcb.153.7.F39
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- Article
The MuSK-BMP pathway maintains myofiber size in slow muscle through regulation of Akt-mTOR signaling.
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- Skeletal Muscle, 2024, v. 14, n. 1, p. 1, doi. 10.1186/s13395-023-00329-9
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- Article
Acetylcholine receptor-aggregating factor is similar to molecules concentrated at neuromuscular junctions.
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- Nature, 1985, v. 315, n. 6020, p. 571, doi. 10.1038/315571a0
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- Article
Aggregates of acetylcholinesterase induced by acetylcholine receptor-aggregating factor.
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- Nature, 1985, v. 315, n. 6020, p. 574, doi. 10.1038/315574a0
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- Article
The FXG: A Presynaptic Fragile X Granule Expressed in a Subset of Developing Brain Circuits.
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- Journal of Neuroscience, 2009, v. 29, n. 5, p. 1514, doi. 10.1523/JNEUROSCI.3937-08.2009
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- Article
Dendrites Contain a Spacing Pattern.
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- Journal of Neuroscience, 2006, v. 26, n. 4, p. 1154, doi. 10.1523/JNEUROSCI.4424-05.2006
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- Article
Visual Experience Regulates Transient Expression and Dendritic Localization of Fragile X Mental Retardation Protein.
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- Journal of Neuroscience, 2004, v. 24, n. 47, p. 10579, doi. 10.1523/JNEUROSCI.2185-04.2004
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- Article
Fragile X granules are a family of axonal ribonucleoprotein particles with circuit-dependent protein composition and m RNA cargos.
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- Journal of Comparative Neurology, 2018, v. 526, n. 1, p. 96, doi. 10.1002/cne.24321
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- Article
Systematic mapping of fragile X granules in the mouse brain reveals a potential role for presynaptic FMRP in sensorimotor functions.
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- Journal of Comparative Neurology, 2012, v. 520, n. 16, p. 3687, doi. 10.1002/cne.23123
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- Article
Biglycan regulates the expression and sarcolemmal localization of dystrobrevin, syntrophin, and nNOS.
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- FASEB Journal, 2006, v. 20, n. 10, p. 1724, doi. 10.1096/fj.05-5124fje
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- Article
Axonal ribosomes and mRNAs associate with fragile X granules in adult rodent and human brains.
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- Human Molecular Genetics, 2017, v. 26, n. 1, p. 192, doi. 10.1093/hmg/ddw381
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- Article
Biglycan binds to α- and γ-sarcoglycan and regulates their expression during development.
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- Journal of Cellular Physiology, 2006, v. 209, n. 2, p. 439, doi. 10.1002/jcp.20740
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- Article
Regulating fragile X gene transcription in the brain and beyond.
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- Journal of Cellular Physiology, 2005, v. 205, n. 2, p. 170, doi. 10.1002/jcp.20401
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- Article
Calcium channels put synapses in their place.
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- 2011
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- Publication type:
- Opinion
Synapses go nucle(ol)ar.
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- Nature Neuroscience, 2007, v. 10, n. 4, p. 399, doi. 10.1038/nn0407-399
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- Article
Internalization of ionotropic glutamate receptors in response to mGluR activation.
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- Nature Neuroscience, 2001, v. 4, n. 11, p. 1079, doi. 10.1038/nn746
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- Article
Dendritic mRNA translation: deciphering the uncoded.
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- Nature Neuroscience, 2000, v. 3, n. 11, p. 1062, doi. 10.1038/80560
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- Article
Building inhibitory synapses: exchange factors getting into the act?
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- Nature Neuroscience, 2000, v. 3, n. 1, p. 5, doi. 10.1038/71080
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- Article
On the Path to a Duchenne Muscular Dystrophy Therapy.
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- Rhode Island Medical Journal, 2013, v. 96, n. 2, p. 22
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- Article
AP-2α selectively regulates fragile X mental retardation-1 gene transcription during embryonic development.
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- Human Molecular Genetics, 2005, v. 14, n. 14, p. 2027, doi. 10.1093/hmg/ddi207
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- Article
Synapses take the rap.
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- Nature, 1995, v. 377, n. 6546, p. 195, doi. 10.1038/377195a0
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- Article
Fragile X Mental Retardation Protein Regulates Olfactory Sensitivity But Not Odorant Discrimination.
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- Chemical Senses, 2015, v. 40, n. 5, p. 345, doi. 10.1093/chemse/bjv019
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- Article
Biglycan Is an Extracellular MuSK Binding Protein Important for Synapse Stability.
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- Journal of Neuroscience, 2012, v. 32, n. 7, p. 2324, doi. 10.1523/JNEUROSCI.4610-11.2012
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- Article