COMPOUNDS AND METHODS FOR MODULATING SMN2

Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle s...

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Bibliographische Detailangaben
Hauptverfasser:	LING, Kar Yun Karen, DRURY, William John, III, PRAKASH, Thazha P, RIGO, Frank, WAN, W. Brad
Format:	Patent
Sprache:	eng ; spa
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival. Se proporcionan compuestos, métodos y composiciones farmacéuticas para modular el ARN y/o proteína de SMN2 en una célula o sujeto. Dichos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma de un trastorno neurodegenerativo. Dichos síntomas incluyen fuerza muscular reducida; incapacidad o capacidad reducida para sentarse erguido, pararse y/o caminar; actividad neuromuscular reducida; actividad eléctrica reducida en uno o más músculos; respiración reducida; incapacidad o capacidad reducida para comer, beber y/o respirar sin ayuda; pérdida de peso o aumento de peso reducido; y/o supervivencia disminuida.