Recent advances in the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Willis, Tracey [verfasserIn] |
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| Format: |
E-Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2021transfer abstract |
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| Umfang: |
5 |
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| Übergeordnetes Werk: |
Enthalten in: Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood - Hinchliff, Gemma L.M. ELSEVIER, 2016, Amsterdam |
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| Übergeordnetes Werk: |
volume:31 ; year:2021 ; number:9 ; pages:359-363 ; extent:5 |
| Links: |
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| DOI / URN: |
10.1016/j.paed.2021.06.005 |
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| 520 | |a Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. | ||
| 520 | |a Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. | ||
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10.1016/j.paed.2021.06.005 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001506.pica (DE-627)ELV055143148 (ELSEVIER)S1751-7222(21)00097-4 DE-627 ger DE-627 rakwb eng 630 VZ 640 VZ 590 VZ 610 VZ 600 VZ 50.70 bkl Willis, Tracey verfasserin aut Recent advances in the treatment of Duchenne muscular dystrophy 2021transfer abstract 5 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. life expectancy Elsevier Duchenne muscular dystrophy Elsevier gene therapy Elsevier clinical trials Elsevier quality of life Elsevier gene editing Elsevier Enthalten in Elsevier Hinchliff, Gemma L.M. ELSEVIER Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood 2016 Amsterdam (DE-627)ELV024210234 volume:31 year:2021 number:9 pages:359-363 extent:5 https://doi.org/10.1016/j.paed.2021.06.005 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U GBV_ILN_40 GBV_ILN_50 GBV_ILN_176 GBV_ILN_181 GBV_ILN_203 GBV_ILN_227 GBV_ILN_235 GBV_ILN_352 GBV_ILN_665 GBV_ILN_677 50.70 Energie: Allgemeines VZ AR 31 2021 9 359-363 5 |
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10.1016/j.paed.2021.06.005 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001506.pica (DE-627)ELV055143148 (ELSEVIER)S1751-7222(21)00097-4 DE-627 ger DE-627 rakwb eng 630 VZ 640 VZ 590 VZ 610 VZ 600 VZ 50.70 bkl Willis, Tracey verfasserin aut Recent advances in the treatment of Duchenne muscular dystrophy 2021transfer abstract 5 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. life expectancy Elsevier Duchenne muscular dystrophy Elsevier gene therapy Elsevier clinical trials Elsevier quality of life Elsevier gene editing Elsevier Enthalten in Elsevier Hinchliff, Gemma L.M. ELSEVIER Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood 2016 Amsterdam (DE-627)ELV024210234 volume:31 year:2021 number:9 pages:359-363 extent:5 https://doi.org/10.1016/j.paed.2021.06.005 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U GBV_ILN_40 GBV_ILN_50 GBV_ILN_176 GBV_ILN_181 GBV_ILN_203 GBV_ILN_227 GBV_ILN_235 GBV_ILN_352 GBV_ILN_665 GBV_ILN_677 50.70 Energie: Allgemeines VZ AR 31 2021 9 359-363 5 |
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10.1016/j.paed.2021.06.005 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001506.pica (DE-627)ELV055143148 (ELSEVIER)S1751-7222(21)00097-4 DE-627 ger DE-627 rakwb eng 630 VZ 640 VZ 590 VZ 610 VZ 600 VZ 50.70 bkl Willis, Tracey verfasserin aut Recent advances in the treatment of Duchenne muscular dystrophy 2021transfer abstract 5 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. life expectancy Elsevier Duchenne muscular dystrophy Elsevier gene therapy Elsevier clinical trials Elsevier quality of life Elsevier gene editing Elsevier Enthalten in Elsevier Hinchliff, Gemma L.M. ELSEVIER Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood 2016 Amsterdam (DE-627)ELV024210234 volume:31 year:2021 number:9 pages:359-363 extent:5 https://doi.org/10.1016/j.paed.2021.06.005 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U GBV_ILN_40 GBV_ILN_50 GBV_ILN_176 GBV_ILN_181 GBV_ILN_203 GBV_ILN_227 GBV_ILN_235 GBV_ILN_352 GBV_ILN_665 GBV_ILN_677 50.70 Energie: Allgemeines VZ AR 31 2021 9 359-363 5 |
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10.1016/j.paed.2021.06.005 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001506.pica (DE-627)ELV055143148 (ELSEVIER)S1751-7222(21)00097-4 DE-627 ger DE-627 rakwb eng 630 VZ 640 VZ 590 VZ 610 VZ 600 VZ 50.70 bkl Willis, Tracey verfasserin aut Recent advances in the treatment of Duchenne muscular dystrophy 2021transfer abstract 5 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. life expectancy Elsevier Duchenne muscular dystrophy Elsevier gene therapy Elsevier clinical trials Elsevier quality of life Elsevier gene editing Elsevier Enthalten in Elsevier Hinchliff, Gemma L.M. ELSEVIER Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood 2016 Amsterdam (DE-627)ELV024210234 volume:31 year:2021 number:9 pages:359-363 extent:5 https://doi.org/10.1016/j.paed.2021.06.005 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U GBV_ILN_40 GBV_ILN_50 GBV_ILN_176 GBV_ILN_181 GBV_ILN_203 GBV_ILN_227 GBV_ILN_235 GBV_ILN_352 GBV_ILN_665 GBV_ILN_677 50.70 Energie: Allgemeines VZ AR 31 2021 9 359-363 5 |
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10.1016/j.paed.2021.06.005 doi /cbs_pica/cbs_olc/import_discovery/elsevier/einzuspielen/GBV00000000001506.pica (DE-627)ELV055143148 (ELSEVIER)S1751-7222(21)00097-4 DE-627 ger DE-627 rakwb eng 630 VZ 640 VZ 590 VZ 610 VZ 600 VZ 50.70 bkl Willis, Tracey verfasserin aut Recent advances in the treatment of Duchenne muscular dystrophy 2021transfer abstract 5 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. life expectancy Elsevier Duchenne muscular dystrophy Elsevier gene therapy Elsevier clinical trials Elsevier quality of life Elsevier gene editing Elsevier Enthalten in Elsevier Hinchliff, Gemma L.M. ELSEVIER Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood 2016 Amsterdam (DE-627)ELV024210234 volume:31 year:2021 number:9 pages:359-363 extent:5 https://doi.org/10.1016/j.paed.2021.06.005 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U GBV_ILN_40 GBV_ILN_50 GBV_ILN_176 GBV_ILN_181 GBV_ILN_203 GBV_ILN_227 GBV_ILN_235 GBV_ILN_352 GBV_ILN_665 GBV_ILN_677 50.70 Energie: Allgemeines VZ AR 31 2021 9 359-363 5 |
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Enthalten in Risky dieting amongst adolescent girls: Associations with family relationship problems and depressed mood Amsterdam volume:31 year:2021 number:9 pages:359-363 extent:5 |
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Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. |
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Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. |
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Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500–5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of improved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials. This review will cover both the updated standards of care and recommended treatments as well as the newer drugs and trials including genetic modification therapies, gene therapy, small molecules to increase the levels of dystrophin related protein and mutation non-specific anti-inflammatory and anti-fibrotic approaches. |
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