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590 results for “muscular dystrophies.”
Supplemental data from: Adrenal suppression from vamorolone and prednisone in Duchenne muscular dystrophy: results from the phase 2b clinical trial
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Data from: Increased Dystrophin Production With Golodirsen in Patients with Duchenne Muscular Dystrophy
<p>Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Methods Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly golodirsen; Part 2 is an ongoing, open-label evaluation. Safety and pharmacokinetics were primary and secondary objectives of Part 1. Primary biological outcome measures of part 2 were blinded exon skipping and dystrophin protein production on muscle biopsies (baseline, Week 48) evaluated, respectively using reverse transcription PCR and western blot and immunohistochemistry. Results Twelve patients were randomized to receive golodirsen (n=8) or placebo (n=4) in Part 1. All from Part 1 plus 13 additional patients received 30 mg/kg golodirsen in Part 2. Safety findings were consistent with those previously observed in pediatric DMD patients. Most of the study drug was excreted within 4 hours following administration. A significant increase in exon 53 skipping was associated with ~16-fold increase over baseline in dystrophin protein expression at Week 48, with a mean percent normal dystrophin protein standard of 1.019% (range, 0.09%-4.30%). Sarcolemmal localization of dystrophin was demonstrated by significantly increased dystrophin positive fibers (Week 48, p<0.001); and a positive correlation (Spearman-r=0.663; p<0.001) between dystrophin protein change from baseline, measured by western blot and immunohistochemistry.</p>
Data from: Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
Objective: We carried out first-in-patient studies of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. Methods: An open-label, multiple-ascending dose study of vamorolone was conducted in 48 boys with Duchenne muscular dystrophy (4 to <7 years, steroid-naïve). Dose levels were 0.25, 0.75, 2.0 and 6.0 mg/kg/day using an oral suspension formulation (12 boys/dose level; 1/3rd to 10x glucocorticoid dose in DMD). Results: Over a 24-week treatment period, oral administration of vamorolone at all doses tested was safe and well-tolerated. The 2.0 mg/kg/day dose group met the primary efficacy outcome of improved muscle function (time to stand; 24 weeks vamorolone treatment versus natural history controls), without evidence of most adverse effects of glucocorticoids. Significant dose-responsive improvements in 10 meter run/walk, and six-minute walk test were observed for 2.0 and 6.0 mg/kg/day dose groups. The morbidity of most concern to many chronic glucocorticoid users, bone loss, was not seen with vamorolone at any dose, as evidenced by serum osteocalcin. Biomarker outcomes for adrenal suppression and insulin resistance were also less impacted in vamorolone-treated DMD patients, relative to published studies of glucocorticoid therapy. Conclusions: Vamorolone demonstrated both efficacy and a reduction in adverse effects in DMD patients compared to traditional glucocorticoids in a 24-week, open-label study. Vamorolone has potential to replace chronic glucocorticoids in many disorders where side effects detract from patient quality of life. Classification of Evidence: This study provides Class II evidence that certain dosages of vamorolone are well tolerated and effective in improving muscle function in DMD.
Data from: Valproic acid is protective in cellular and worm models of oculopharyngeal muscular dystrophy
Objective: To explore valproic acid (VPA) as a potentially beneficial drug in cellular and worm models of oculopharyngeal muscular dystrophy (OPMD). Methods: Using a combination of live cell imaging and biochemical measures, we evaluated the potential protective effect of VPA in a stable C2C12 muscle cell model of OPMD, in lymphoblastoid cell lines derived from patients with OPMD and in a transgenic Caenorhabditis elegans OPMD model expressing human mutant PABPN1. Results: We demonstrated that VPA protects against the toxicity of mutant PABPN1. Of note, we found that VPA confers its long-term protective effects on C2C12 cell survival, proliferation, and differentiation by increasing the acetylated level of histones. Furthermore, VPA enhances the level of histone acetylation in lymphoblastoid cell lines derived from patients with OPMD. Moreover, treatment of nematodes with moderate concentrations of VPA significantly improved the motility of the PABPN-13 Alanines worms. Conclusions: Our results suggest that VPA helps to counteract OPMD-related phenotypes in the cellular and C elegans disease models.
Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
ClinicalTrials.gov study NCT02814110. IPD Sharing: UNDECIDED. Countries: 1. Publications: 3.
Motor Imagery in Duchenne Muscular Dystrophy
ClinicalTrials.gov study NCT05559710. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Transition to Adulthood in People With Muscular Dystrophy
ClinicalTrials.gov study NCT07101185. IPD Sharing: NO. Countries: 1. Publications: 1.
Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
ClinicalTrials.gov study NCT06773988. IPD Sharing: NO. Countries: 1. Publications: 3.
High Intensity Training in Patients With Facioscapulohumeral Muscular Dystrophy
ClinicalTrials.gov study NCT02159963. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Examination of Operative Approach in pwFSHD (Patient With Facioscapulohumeral Muscular Dystrophy)
ClinicalTrials.gov study NCT05022355. IPD Sharing: Not stated. Countries: 1. Publications: 14.
Comprehensive Study of Duchenne Muscular Dystrophy at Sohag University Hospital
ClinicalTrials.gov study NCT05029232. IPD Sharing: YES. Countries: 1. Publications: 3.
Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov study NCT00468832. IPD Sharing: Not stated. Countries: 8. Publications: 2.
Long-Term Development of Muscular Dystrophy Outcome Assessments
ClinicalTrials.gov study NCT05989620. IPD Sharing: NO. Countries: 1. Publications: 21.
Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy
ClinicalTrials.gov study NCT05464446. IPD Sharing: UNDECIDED. Countries: 1. Publications: 1.
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
ClinicalTrials.gov study NCT02050776. IPD Sharing: Not stated. Countries: 1. Publications: 1.
1 Year MRI Followup in Facioscapulohumeral Muscular Dystrophy
ClinicalTrials.gov study NCT02159612. IPD Sharing: Not stated. Countries: 1. Publications: 1.
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
ClinicalTrials.gov study NCT04585464. IPD Sharing: NO. Countries: 1. Publications: 1.
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
ClinicalTrials.gov study NCT03490214. IPD Sharing: NO. Countries: 1. Publications: 1.
Effectiveness of Aquatic Therapy in Children With Duchenne and Becker Muscular Dystrophy
ClinicalTrials.gov study NCT06186310. IPD Sharing: NO. Countries: 1. Publications: 1.
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
ClinicalTrials.gov study NCT04120168. IPD Sharing: NO. Countries: 1. Publications: 1.
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