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About 4,503 results

ALLMedicine™ Duchenne Muscular Dystrophy Center

Research & Reviews  1,679 results

A new therapeutic effect of fenofibrate in Duchenne muscular dystrophy: The promotion o...
https://doi.org/10.1111/bph.15678
British Journal of Pharmacology; Sun Z, Xu D et. al.

Sep 24th, 2021 - Duchenne muscular dystrophy (DMD) is a degenerative muscle disease with no effective drug treatment. This study investigated the positive effects of fenofibrate on dystrophic muscles. Myostatin expression in serum and muscle tissue of DMD patients...

A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystr...
https://doi.org/10.1089/hum.2021.29179.ddu
Human Gene Therapy; Duan D

Sep 24th, 2021 - A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients.|2021|Duan D,|

Articular cartilage degeneration and bone adaptation due to lack of dystrophin in mice.
https://doi.org/10.1007/s00774-021-01270-x
Journal of Bone and Mineral Metabolism; Dos Santos JF, Lazzarin MC et. al.

Sep 23rd, 2021 - Duchenne muscular dystrophy is caused by the absence of dystrophin. This study aimed to investigate femoral morphological characteristics of lack of dystrophin in MDX mice, considering that this model, different from DMD patient, is not influenced...

The PPLD has advantages over conventional regression methods in application to moderate...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8457474
PloS One; Vieland VJ, Seok SC

Sep 23rd, 2021 - In earlier work, we have developed and evaluated an alternative approach to the analysis of GWAS data, based on a statistic called the PPLD. More recently, motivated by a GWAS for genetic modifiers of the X-linked Mendelian disorder Duchenne Muscu...

Emotional behavior and brain anatomy of the mdx52 mouse model of Duchenne muscular dyst...
https://doi.org/10.1242/dmm.049028
Disease Models & Mechanisms; Saoudi A, Zarrouki F et. al.

Sep 22nd, 2021 - The exon-52-deleted mdx52 mouse is a critical model of Duchenne muscular dystrophy (DMD), as it features a deletion in a hotspot region of the DMD gene, frequently mutated in patients. Deletion of exon 52 impedes expression of several brain dystro...

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Guidelines  4 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 3rd, 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...

Consensus guidelines on the use of bisphosphonate therapy in children and adolescents.
https://doi.org/10.1111/jpc.13768
Journal of Paediatrics and Child Health; Simm PJ, Biggin A et. al.

Mar 6th, 2018 - Bisphosphonate therapy is the mainstay of pharmacological intervention in young people with skeletal fragility. The evidence of its use in a variety of conditions remains limited despite over three decades of clinical experience. On behalf of the ...

Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the Nation...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4573596
Circulation McNally EM, Kaltman JR et. al.

May 6th, 2015 - Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy.|2015|McNally EM,Kaltman JR,Benson DW,Canter CE,Cripe LH,|therapeuti...

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmac...
https://doi.org/10.1016/S1474-4422(09)70271-6
The Lancet. Neurology; Bushby K, Finkel R et. al.

Dec 1st, 2009 - Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600-6000 live male births. Although guidelines are available for various aspects of DMD, comprehensive clinical care recommendations do not exist. The US Centers...

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Drugs  8 results see all →

News  108 results

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

Feb 25th, 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 25th, 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.medscape.com/viewarticle/939949

Oct 28th, 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.staging.medscape.com/viewarticle/939949

Oct 28th, 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.medscape.com/viewarticle/935624

Aug 12th, 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  2 results see all →