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About 20,724 results

ALLMedicine™ Muscular Dystrophy Center

Research & Reviews  10,059 results

Over three decades of natural history of limb girdle muscular dystrophy type R1/2A and ...
https://doi.org/10.1016/j.nmd.2021.02.018
Neuromuscular Disorders : NMD; LoMauro A, Gandossini S et. al.

Apr 10th, 2021 - We aimed to describe the natural history of Limb Girdle Muscular Dystrophy type 2A and 2B over more than three decades by considering muscular strength, motor, cardiac and respiratory function. 428 visits of nineteen 2A and twenty 2B patients were...

Defective dystrophic thymus determines degenerative changes in skeletal muscle.
https://doi.org/10.1038/s41467-021-22305-x 10.1136/bmj.317.7164.991 10.1007/s13539-012-0074-6 10.1006/clim.2000.4966 10.1172/JCI37662 10.1056/NEJMoa1000228 10.1080/21675511.2015.1010966 10.1038/mt.2016.162 10.1002/path.2213 10.1016/j.cell.2013.10.054 10.1111/j.1365-2567.2008.02992.x 10.1084/jem.20121412 10.4049/jimmunol.180.4.2299 10.1002/eji.201545844 10.1016/0165-5728(95)00010-Y 10.1002/eji.201142305 10.1016/j.coi.2013.06.002 10.1038/nri3667 10.1172/JCI30248 10.1182/blood-2008-09-181255 10.5115/acb.2011.44.1.14 10.1016/j.aucc.2018.10.007 10.1074/jbc.M808302200 10.1016/j.bcp.2006.08.007 10.1038/nri.2017.52 10.1093/intimm/11.8.1357 10.1158/0008-5472.CAN-05-1602 10.1038/cddis.2012.159 10.1080/15548627.2016.1144005 10.1016/j.biocel.2004.02.005 10.1038/nature07208 10.1016/S0076-6879(08)03612-4 10.1038/icb.2014.81 10.1016/j.immuni.2008.08.010 10.1038/labinvest.3780092 10.1073/pnas.81.3.886 10.1084/jem.138.5.1044 10.1016/j.cell.2016.04.014 10.1016/0092-8674(88)90463-1 10.1016/j.jmb.2008.08.087 10.1007/s12013-011-9175-7 10.1016/j.jmb.2007.11.049 10.1093/hmg/ddr015 10.1093/hmg/ddn358 10.1093/hmg/11.3.263 10.1002/path.4801 10.1016/j.cytogfr.2006.09.006 10.1016/j.molcel.2013.06.020 10.1093/hmg/ddt536 10.1006/jmcc.1999.1018 10.1016/j.molcel.2011.04.029 10.1371/journal.pone.0030063 10.1111/j.1749-6632.2012.06738.x 10.1371/journal.pone.0117306 10.1074/jbc.M802585200 10.1111/trf.12334 10.1371/journal.pone.0108319 10.1146/annurev.immunol.23.021704.115601 10.1152/ajpheart.00413.2018 10.15252/emmm.201403967 10.1093/hmg/ddn376 10.1126/scitranslmed.3009925 10.1371/journal.pone.0031515 10.1038/icb.2008.87 10.1038/nri3155 10.1016/j.immuni.2016.04.018 10.4049/jimmunol.172.4.2067 10.1371/journal.pgen.1005777 10.1016/j.jaut.2017.09.006 10.1507/endocrj.49.625 10.1016/j.coph.2010.04.015 10.1172/JCI200421134 10.1007/s00360-019-01253-5 10.1113/jphysiol.2011.206193 10.1096/fj.04-2364com 10.1080/15384101.2018.1482150 10.4161/auto.7.5.15102 10.1038/jid.2012.492 10.1016/j.stem.2007.09.016 10.3389/fonc.2015.00052 10.1007/s00018-009-8652-2 10.1038/srep38371 10.1074/jbc.M400674200 10.1371/journal.pone.0035273 10.1016/j.cell.2015.10.013
Nature Communications; Farini A, Sitzia C et. al.

Apr 9th, 2021 - In Duchenne muscular dystrophy (DMD), sarcolemma fragility and myofiber necrosis produce cellular debris that attract inflammatory cells. Macrophages and T-lymphocytes infiltrate muscles in response to damage-associated molecular pattern signallin...

Alleviation of masticatory disturbance with an occlusal splint in a Duchenne muscular d...
https://doi.org/10.1111/scd.12594
Special Care in Dentistry : Official Publication of the A... Fukumoto Y, Miyama T

Apr 7th, 2021 - To present an occlusal splint effective for alleviating masticatory disturbance in Duchenne muscular dystrophy (DMD). A 13-year-old male DMD patient with masticatory disturbance presented with an open bite, with occlusal contact only between the f...

Pharmacological activation of SERCA ameliorates dystrophic phenotypes in dystrophin-def...
https://doi.org/10.1093/hmg/ddab100
Human Molecular Genetics; Nogami K, Maruyama Y et. al.

Apr 6th, 2021 - Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by progressive muscular weakness due to the loss of dystrophin. Extracellular Ca2+ flows into the cytoplasm through membrane tears in dystrophin-deficient myofibers, w...

CRISPR Technologies for the Treatment of Duchenne Muscular Dystrophy.
https://doi.org/10.1016/j.ymthe.2021.04.002
Molecular Therapy : the Journal of the American Society O... Choi E, Koo T

Apr 6th, 2021 - The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat ...

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Guidelines  23 results

Respiratory Management of the Patient With Duchenne Muscular Dystrophy.
https://doi.org/10.1542/peds.2018-0333H
Pediatrics Sheehan DW, Birnkrant DJ et. al.

Oct 2nd, 2018 - In 2010, Care Considerations for Duchenne Muscular Dystrophy, sponsored by the Centers for Disease Control and Prevention, was published in Lancet Neurology, and in 2018, these guidelines were updated. Since the publication of the first set of gui...

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency man...
https://linkinghub.elsevier.com/retrieve/pii/S1474442218300267

Apr 30th, 2018 - Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to management across a range of health-care specialties. In part 3 of this upda.

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone ...
https://linkinghub.elsevier.com/retrieve/pii/S1474442218300255

Mar 31st, 2018 - A coordinated, multidisciplinary approach to care is essential for optimum management of the primary manifestations and secondary complications of Duchenne muscular dystrophy (DMD). Contemporary care has been shaped by the availability of more sen.

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscula...
https://linkinghub.elsevier.com/retrieve/pii/S1474442218300243

Feb 28th, 2018 - Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more antici.

Duchenne Muscular Dystrophy (DMD)
https://www.mda.org/disease/duchenne-muscular-dystrophy/medical-management
Muscular Dystrophy Association (USA)

Jan 22nd, 2018 - Thanks to advances in many areas of medicine, such as cardiology and pulmonology, people with Duchenne muscular dystrophy in the 21st century are living longer than in previous decades, often well into adulthood. The use of available treatments ca.

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Drugs  15 results see all →

Clinicaltrials.gov  10,339 results

Over three decades of natural history of limb girdle muscular dystrophy type R1/2A and ...
https://doi.org/10.1016/j.nmd.2021.02.018
Neuromuscular Disorders : NMD; LoMauro A, Gandossini S et. al.

Apr 10th, 2021 - We aimed to describe the natural history of Limb Girdle Muscular Dystrophy type 2A and 2B over more than three decades by considering muscular strength, motor, cardiac and respiratory function. 428 visits of nineteen 2A and twenty 2B patients were...

Defective dystrophic thymus determines degenerative changes in skeletal muscle.
https://doi.org/10.1038/s41467-021-22305-x 10.1136/bmj.317.7164.991 10.1007/s13539-012-0074-6 10.1006/clim.2000.4966 10.1172/JCI37662 10.1056/NEJMoa1000228 10.1080/21675511.2015.1010966 10.1038/mt.2016.162 10.1002/path.2213 10.1016/j.cell.2013.10.054 10.1111/j.1365-2567.2008.02992.x 10.1084/jem.20121412 10.4049/jimmunol.180.4.2299 10.1002/eji.201545844 10.1016/0165-5728(95)00010-Y 10.1002/eji.201142305 10.1016/j.coi.2013.06.002 10.1038/nri3667 10.1172/JCI30248 10.1182/blood-2008-09-181255 10.5115/acb.2011.44.1.14 10.1016/j.aucc.2018.10.007 10.1074/jbc.M808302200 10.1016/j.bcp.2006.08.007 10.1038/nri.2017.52 10.1093/intimm/11.8.1357 10.1158/0008-5472.CAN-05-1602 10.1038/cddis.2012.159 10.1080/15548627.2016.1144005 10.1016/j.biocel.2004.02.005 10.1038/nature07208 10.1016/S0076-6879(08)03612-4 10.1038/icb.2014.81 10.1016/j.immuni.2008.08.010 10.1038/labinvest.3780092 10.1073/pnas.81.3.886 10.1084/jem.138.5.1044 10.1016/j.cell.2016.04.014 10.1016/0092-8674(88)90463-1 10.1016/j.jmb.2008.08.087 10.1007/s12013-011-9175-7 10.1016/j.jmb.2007.11.049 10.1093/hmg/ddr015 10.1093/hmg/ddn358 10.1093/hmg/11.3.263 10.1002/path.4801 10.1016/j.cytogfr.2006.09.006 10.1016/j.molcel.2013.06.020 10.1093/hmg/ddt536 10.1006/jmcc.1999.1018 10.1016/j.molcel.2011.04.029 10.1371/journal.pone.0030063 10.1111/j.1749-6632.2012.06738.x 10.1371/journal.pone.0117306 10.1074/jbc.M802585200 10.1111/trf.12334 10.1371/journal.pone.0108319 10.1146/annurev.immunol.23.021704.115601 10.1152/ajpheart.00413.2018 10.15252/emmm.201403967 10.1093/hmg/ddn376 10.1126/scitranslmed.3009925 10.1371/journal.pone.0031515 10.1038/icb.2008.87 10.1038/nri3155 10.1016/j.immuni.2016.04.018 10.4049/jimmunol.172.4.2067 10.1371/journal.pgen.1005777 10.1016/j.jaut.2017.09.006 10.1507/endocrj.49.625 10.1016/j.coph.2010.04.015 10.1172/JCI200421134 10.1007/s00360-019-01253-5 10.1113/jphysiol.2011.206193 10.1096/fj.04-2364com 10.1080/15384101.2018.1482150 10.4161/auto.7.5.15102 10.1038/jid.2012.492 10.1016/j.stem.2007.09.016 10.3389/fonc.2015.00052 10.1007/s00018-009-8652-2 10.1038/srep38371 10.1074/jbc.M400674200 10.1371/journal.pone.0035273 10.1016/j.cell.2015.10.013
Nature Communications; Farini A, Sitzia C et. al.

Apr 9th, 2021 - In Duchenne muscular dystrophy (DMD), sarcolemma fragility and myofiber necrosis produce cellular debris that attract inflammatory cells. Macrophages and T-lymphocytes infiltrate muscles in response to damage-associated molecular pattern signallin...

Alleviation of masticatory disturbance with an occlusal splint in a Duchenne muscular d...
https://doi.org/10.1111/scd.12594
Special Care in Dentistry : Official Publication of the A... Fukumoto Y, Miyama T

Apr 7th, 2021 - To present an occlusal splint effective for alleviating masticatory disturbance in Duchenne muscular dystrophy (DMD). A 13-year-old male DMD patient with masticatory disturbance presented with an open bite, with occlusal contact only between the f...

Pharmacological activation of SERCA ameliorates dystrophic phenotypes in dystrophin-def...
https://doi.org/10.1093/hmg/ddab100
Human Molecular Genetics; Nogami K, Maruyama Y et. al.

Apr 6th, 2021 - Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by progressive muscular weakness due to the loss of dystrophin. Extracellular Ca2+ flows into the cytoplasm through membrane tears in dystrophin-deficient myofibers, w...

CRISPR Technologies for the Treatment of Duchenne Muscular Dystrophy.
https://doi.org/10.1016/j.ymthe.2021.04.002
Molecular Therapy : the Journal of the American Society O... Choi E, Koo T

Apr 6th, 2021 - The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat ...

see more →

News  259 results

FDA OKs First 'Targeted Treatment' for Rare DMD Mutation
https://www.medscape.com/viewarticle/946507

Feb 24th, 2021 - The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne muscular dystrophy (DMD) plus a rare DMD mutation, the agency ...

After 10 Months of Quarantine, Chronically Ill Young German Jumps Vaccine Queue
https://www.medscape.com/viewarticle/944541

Jan 24th, 2021 - NIEDERBREITBACH, Germany (Reuters) - Since the outbreak of the coronavirus pandemic, chronically ill 30-year-old Benni Over has lived in strict domestic quarantine in the German resort town of Niederbreitbach with his parents, who look after him a...

Ataluren Delays Disease Milestones in Patients With Nonsense Mutation DMD
https://www.staging.medscape.com/viewarticle/939949

Oct 27th, 2020 - Long-term treatment with ataluren delays loss of ambulation and may delay decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD), according to study results presented at the 2020 CNS-ICNA Conjoint Meet...

Ataluren Delays Disease Milestones in Patients With Nonsense Mutation DMD
https://www.medscape.com/viewarticle/939949

Oct 27th, 2020 - Long-term treatment with ataluren delays loss of ambulation and may delay decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD), according to study results presented at the 2020 CNS-ICNA Conjoint Meet...

FDA OKs Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy
https://www.staging.medscape.com/viewarticle/935624

Aug 11th, 2020 - The US Food and Drug Administration (FDA) has approved viltolarsen (Viltepso, NS Pharma), the second drug therapy for Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 53 skipping. The FDA approved golodirsen (Vyon...

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Patient Education  29 results see all →