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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1989 2
2003 1
2005 2
2007 1
2011 1
2012 1
2013 3
2016 2
2017 2
2018 7
2019 3
2020 2
2021 5
2022 5
2023 4
2024 2

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40 results

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Page 1
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy.
Li Y, Zeng H, Wei Y, Ma X, He Z. Li Y, et al. Hum Gene Ther. 2023 Mar;34(5-6):180-191. doi: 10.1089/hum.2022.189. Hum Gene Ther. 2023. PMID: 36762938 Review.
It is one of the most common genetic causes of infant mortality and is characterized by muscle weakness, loss of ambulation, and respiratory failure. SMA is primarily caused by a homozygous deletion or mutation of the survival motor neuron 1 (SMN1) gene. ...Onasemno …
It is one of the most common genetic causes of infant mortality and is characterized by muscle weakness, loss of ambulation, a …
Inclusion Body Myositis: Update on Pathogenesis and Treatment.
Naddaf E, Barohn RJ, Dimachkie MM. Naddaf E, et al. Neurotherapeutics. 2018 Oct;15(4):995-1005. doi: 10.1007/s13311-018-0658-8. Neurotherapeutics. 2018. PMID: 30136253 Free PMC article. Review.
It is characterized by progressive asymmetric weakness predominantly affecting the quadriceps and/or finger flexors. Loss of ambulation and dysphagia are major complications of the disease. ...Better understanding of the disease pathogenesis led to the identificatio …
It is characterized by progressive asymmetric weakness predominantly affecting the quadriceps and/or finger flexors. Loss of ambul …
The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review.
Ryder S, Leadley RM, Armstrong N, Westwood M, de Kock S, Butt T, Jain M, Kleijnen J. Ryder S, et al. Orphanet J Rare Dis. 2017 Apr 26;12(1):79. doi: 10.1186/s13023-017-0631-3. Orphanet J Rare Dis. 2017. PMID: 28446219 Free PMC article. Review.
A study of adult DMD patients at a centre in France found median survival for those born between 1970 and 1994 was 40.95 years compared to 25.77 years for those born between 1955 and 1969. Loss of ambulation occurred at a median age of 12 and ventilation starts at a …
A study of adult DMD patients at a centre in France found median survival for those born between 1970 and 1994 was 40.95 years compared to 2 …
Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy.
Aslesh T, Yokota T. Aslesh T, et al. Cells. 2022 Jan 26;11(3):417. doi: 10.3390/cells11030417. Cells. 2022. PMID: 35159227 Free PMC article. Review.
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder and one of the most common genetic causes of infant death. It is characterized by progressive weakness of the muscles, loss of ambulation, and death from respiratory complications. SM …
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder and one of the most common genetic causes of infant death …
Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis.
Weber FJ, Latshang TD, Blum MR, Kohler M, Wertli MM. Weber FJ, et al. Muscle Nerve. 2022 Oct;66(4):462-470. doi: 10.1002/mus.27682. Epub 2022 Aug 6. Muscle Nerve. 2022. PMID: 35860996 Free PMC article.
INTRODUCTION/AIMS: Prognostic factors in Duchenne muscular dystrophy (DMD) predict the disease course and may help individualize patient care. The aim was to summarize the evidence on prognostic factors that may support treatment decisions. METHODS: We searched six databas …
INTRODUCTION/AIMS: Prognostic factors in Duchenne muscular dystrophy (DMD) predict the disease course and may help individualize patient …
Evolving Therapeutic Options for the Treatment of Duchenne Muscular Dystrophy.
D'Ambrosio ES, Mendell JR. D'Ambrosio ES, et al. Neurotherapeutics. 2023 Oct;20(6):1669-1681. doi: 10.1007/s13311-023-01423-y. Epub 2023 Sep 6. Neurotherapeutics. 2023. PMID: 37673849 Free article. Review.
It is caused by mutations in the DMD gene, leading to reduced or absent expression of the dystrophin protein. Clinically, this results in loss of ambulation, cardiomyopathy, respiratory failure, and eventually death. ...In this review, we highlight the most up-to-da …
It is caused by mutations in the DMD gene, leading to reduced or absent expression of the dystrophin protein. Clinically, this results in …
Advances in the diagnosis and management of cardiomyopathy in Duchenne muscular dystrophy.
Hor KN, Mah ML, Johnston P, Cripe TP, Cripe LH. Hor KN, et al. Neuromuscul Disord. 2018 Sep;28(9):711-716. doi: 10.1016/j.nmd.2018.06.014. Epub 2018 Jul 6. Neuromuscul Disord. 2018. PMID: 30064893 Review.
Patients with Duchenne muscular dystrophy suffer debilitating muscle destruction, resulting in loss of ambulation, diminished respiratory function, gastrointestinal disturbances and cardiomyopathy. Although it is the most common cause of death in these patients, car …
Patients with Duchenne muscular dystrophy suffer debilitating muscle destruction, resulting in loss of ambulation, diminished …
Bone Health and Osteoporosis Management of the Patient With Duchenne Muscular Dystrophy.
Ward LM, Hadjiyannakis S, McMillan HJ, Noritz G, Weber DR. Ward LM, et al. Pediatrics. 2018 Oct;142(Suppl 2):S34-S42. doi: 10.1542/peds.2018-0333E. Pediatrics. 2018. PMID: 30275247 Free PMC article. Review.
Duchenne muscular dystrophy is associated with an increased risk of bone fragility due to the adverse effects of prolonged glucocorticoid therapy and progressive muscle weakness on bone strength. Osteoporosis manifests clinically as low-trauma long-bone and vertebral fract …
Duchenne muscular dystrophy is associated with an increased risk of bone fragility due to the adverse effects of prolonged glucocorticoid …
Clinical Manifestations and Overall Management Strategies for Duchenne Muscular Dystrophy.
Tsuda T. Tsuda T. Methods Mol Biol. 2018;1687:19-28. doi: 10.1007/978-1-4939-7374-3_2. Methods Mol Biol. 2018. PMID: 29067653 Review.
The progressive destruction of systemic musculature and myocardium causes affected patients to develop multiple organ disabilities, including loss of ambulation, physical immobility, neuromuscular scoliosis, joint contracture, restrictive lung disease, obstructive s …
The progressive destruction of systemic musculature and myocardium causes affected patients to develop multiple organ disabilities, includin …
Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1.
McDonald CM, Mayer OH, Hor KN, Miller D, Goemans N, Henricson EK, Marden JR, Freimark J, Lane H, Zhang A, Frean M, Trifillis P, Koladicz K, Signorovitch J; PRO-DMD-01 consortium investigators. McDonald CM, et al. J Neuromuscul Dis. 2023;10(1):67-79. doi: 10.3233/JND-221575. J Neuromuscul Dis. 2023. PMID: 36565131 Free PMC article.
BACKGROUND: Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy (DMD) after loss of ambulation is limited. OBJECTIVE: Characterize and compare disease progression by steroid treatment (prednisone, deflazacort, or no steroids) among non-ambu …
BACKGROUND: Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy (DMD) after loss of ambulation is li …
40 results