- |||||||||| prednisone / Generic mfg., Agamree (vamorolone) / Santhera
Retrospective data, Review, Journal: Vamorolone Versus Traditional Glucocorticoids in Duchenne Muscular Dystrophy: A Review and Meta-Analysis of Efficacy and Safety. (Pubmed Central) - Jun 27, 2025
Safety outcomes showed enhanced height percentile (MD = 16.28, 95% CI = 6.31-26.25, P = .001) and bone biomarkers (osteocalcin: MD = 15.68, P < .00001; P1NP: MD = 158.34, P < .00001), with no significant body mass index-z score difference. Vamorolone offers comparable efficacy with traditional glucocorticoids in DMD, with improved motor function and safer profiles in growth and bone health, suggesting its potential to transform DMD management.
- |||||||||| FDA event, Journal: Pharmacological Insights on USFDA-Approved Novel Drug Therapies in the Year 2023. (Pubmed Central) - Jun 4, 2025
The novel drug therapies approved by the USFDA hold significant potential to enhance the patient's care by providing advanced treatment modalities. This manuscript, reporting the comprehensive description of therapeutic aspects of the mentioned new drug therapies, underscores the commitment of the pharmaceutical sector to address the unmet medical needs and reshape the landscape of the healthcare service system by instilling optimism among patients and healthcare providers.
- |||||||||| Current Challenges and Solutions for Drug Loss in Duchenne Muscular Dystrophy (Venue 06 (Osaka International Convention Center, 10F, Conference Room 1008)) - May 8, 2025 - Abstract #JSNE2025JSNE_1827;
This manuscript, reporting the comprehensive description of therapeutic aspects of the mentioned new drug therapies, underscores the commitment of the pharmaceutical sector to address the unmet medical needs and reshape the landscape of the healthcare service system by instilling optimism among patients and healthcare providers. As of March 2023, 143 items fall under drug loss, including DMD treatments Vyondys53
- |||||||||| Agamree (vamorolone) / Santhera
Retrospective data, Journal: Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy. (Pubmed Central) - Apr 16, 2025
As of March 2023, 143 items fall under drug loss, including DMD treatments Vyondys53 The results of our systematic review and meta-analyis are suggestive of improved efficacy and safety of vamorolone for DMD compared to standard glucocorticosteroids but the external validity of these findings as well as the medication's long-term effects remain to be determined.
- |||||||||| Review, Journal: Early Cardiac Dysfunction in Duchenne Muscular Dystrophy: A Case Report and Literature Update. (Pubmed Central) - Feb 26, 2025
This case report presents a rare instance of early-onset cardiac involvement in a 3-year-old male with a confirmed deletion in exon 55 of the dystrophin gene...Genetic mechanisms and genotype-phenotype correlations related to cardiac involvement were reviewed, highlighting emerging therapies such as exon skipping, vamorolone, ifetroban, and rimeporide...While current DMD care standards improve survival, optimizing management through early intervention and novel therapies remains essential. Further research is needed to better understand genotype-phenotype correlations and improve cardiac outcomes for patients with DMD.
- |||||||||| Review, Journal: The latest developments in synthetic approaches to duchenne muscular dystrophy. (Pubmed Central) - Feb 3, 2025
Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options...The accelerated FDA review process has enabled faster approval of new medications; however many have provided minimal clinical benefit to patients. Despite these challenges, continued drug development and innovative research offer hope to patients.
- |||||||||| Agamree (vamorolone) / Santhera
Enrollment closed: A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) (clinicaltrials.gov) - Dec 27, 2024
P2, N=39, Active, not recruiting,
Despite these challenges, continued drug development and innovative research offer hope to patients. Recruiting --> Active, not recruiting
- |||||||||| Agamree (vamorolone) / Santhera
Journal: Metabolic Characterization of Vamorolone in Human Liver Microsomes: Implications for Anti-Doping. (Pubmed Central) - Oct 31, 2024
The findings not only enhance the understanding of its metabolic mechanisms but also offer a scientific basis for evaluating its safety and efficacy in sports medicine. Meanwhile, these discoveries can contribute to better regulation and control of Vamorolone's use in competitive sports, ensuring fairness in competitions.
- |||||||||| Agamree (vamorolone) / Santhera
Review, Journal: Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review. (Pubmed Central) - Oct 28, 2024
According to our results, vamorolone offers a statistically and clinically significant benefit in the management of Duchenne muscular dystrophy, with fewer side effects than glucocorticoids. However, the number of studies limits the interpretability and generalisability of these data, requiring more studies with more participants to perform a meta-analysis.
- |||||||||| Agamree (vamorolone) / Santhera
Review, Journal, Adverse events: Corticosteroids for the treatment of duchenne muscular dystrophy: a safety review. (Pubmed Central) - Oct 10, 2024
When selecting a medication for a patient, the decision-making process will most likely rely on the minor differences in the adverse effect profiles. Whichever medication is utilized will surely be a part of a larger regimen that includes other novel therapeutic agents.
- |||||||||| Agamree (vamorolone) / Santhera
Trial completion date, Trial primary completion date: A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) (clinicaltrials.gov) - Aug 19, 2024
P2, N=39, Recruiting,
Together, these results implicate intramyocellular Ca2+ leak as a critical driver of dysferlinopathic phenotype and validate the myobundle system as a platform to study LGMD2B pathogenesis. Trial completion date: Jan 2025 --> Jun 2025 | Trial primary completion date: Jan 2025 --> Jun 2025
- |||||||||| BGP-15 - N / Gene, Agamree (vamorolone) / Santhera
Journal: CAS3D: 3D quantitative morphometry on Second Harmonic Generation image volumes from single skeletal muscle fibers. (Pubmed Central) - Jul 23, 2024
Our method reliably reproduced the results of the previous work and improved the standard deviation of the cosine angle sum detection in all sample groups from a mean of 0.03 to 0.008. This improvement is achieved by applying analysis algorithms to the whole volumetric images in 3D in contrast to the previously common method of slice-wise XY analysis.
- |||||||||| Translarna (ataluren) / PTC Therap, Agamree (vamorolone) / Santhera
Journal: The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report. (Pubmed Central) - Mar 11, 2024
The meeting emphasized the need for early diagnosis, evidence-based guidelines, and comprehensive care and support for affected children and their families. Further research, collaboration, and the development of consensus guidelines are needed to improve early diagnosis, treatment, and outcomes in this population.
- |||||||||| Emflaza (deflazacort) / Marathon, PTC Therap, Agamree (vamorolone) / Santhera
Journal: Comparison of pharmaceutical properties and biological activity of prednisolone, deflazacort, and vamorolone in DMD disease models. (Pubmed Central) - Jan 22, 2024
The corticosteroids prednisone/prednisolone and deflazacort are used to treat DMD as the standard of care; however, only deflazacort is FDA approved for DMD...The results of these studies indicate that efficacious doses of vamorolone, are associated with similar side effects as seen with other corticosteroids. Further, because vamorolone is not a strong P-gp substrate, vamorolone distributes into the CNS increasing the potential CNS side-effects.
- |||||||||| Agamree (vamorolone) / Santhera
Review, Journal: Vamorolone: First Approval. (Pubmed Central) - Jan 17, 2024
Vamorolone was approved in October 2023 for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older in the USA and received a positive opinion in the EU in October 2023 for the treatment of DMD in patients 4 years of age and older. This article summarizes the milestones in the development of vamorolone leading to this first approval for DMD.
- |||||||||| Agamree (vamorolone) / Santhera
Journal: Vamorolone. (Pubmed Central) - Dec 28, 2023
This article summarizes the milestones in the development of vamorolone leading to this first approval for DMD. No abstract available
- |||||||||| Agamree (vamorolone) / Santhera
Journal: Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy. (Pubmed Central) - Nov 14, 2023
Of particular interest to clarify is the optimal clinical dose and other aspects of vamorolone that are proposed to provide additional benefits for membranes of dystrophic muscle: to stabilise and protect the sarcolemma from damage and enhance repair. The use of vamorolone (and other glucocorticoids) needs to be evaluated in terms of overall long-term efficacy and cost, and also in comparison with many candidate non-steroidal drugs with anti-inflammatory and other benefits for DMD.
- |||||||||| Agamree (vamorolone) / Santhera
Trial completion date, Trial primary completion date: A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Nov 9, 2023
P2, N=54, Recruiting,
The use of vamorolone (and other glucocorticoids) needs to be evaluated in terms of overall long-term efficacy and cost, and also in comparison with many candidate non-steroidal drugs with anti-inflammatory and other benefits for DMD. Trial completion date: Dec 2023 --> Mar 2024 | Trial primary completion date: Dec 2023 --> Mar 2024
- |||||||||| Review, Journal: Advances in Dystrophinopathy Diagnosis and Therapy. (Pubmed Central) - Oct 4, 2023
Other antisense oligonucleotide drugs in the pipeline include casimersen for exon 45, suvodirsen for exon 51, and golodirsen for exon 53 skipping. Advances in the diagnosis and therapy of dystrophinopathies offer new perspectives for their early discovery and care.
- |||||||||| Review, Journal: Single-neuron neurodegeneration as a degenerative model for Parkinson's disease. (Pubmed Central) - Sep 18, 2023
In conclusion, based on the hypothesis that the neurodegenerative process of idiopathic Parkinson's disease corresponds to a single-neuron neurodegeneration model, we must search for molecules that increase the expression of the neuroprotective enzymes DT-diaphorase and glutathione transferase M2-2. It has been observed that the activation of the Kelch-like ECH-associated protein 1/nuclear factor (erythroid-derived 2)-like 2 pathway is associated with the transcriptional activation of the DT-diaphorase and glutathione transferase genes.
- |||||||||| Catena (idebenone) / Santhera, Chiesi
Review, Journal: Current and Future Landscape in Genetic Therapies for Leber Hereditary Optic Neuropathy. (Pubmed Central) - Aug 11, 2023
This manuscript discusses the phenotype and genotype of the LHON disease with complexities and peculiarities such as incomplete penetrance and gender bias, which have challenged the therapies in development emphasising the most recent use of gene therapy. Furthermore, we review the latest results of the three clinical trials based on adeno-associated viral (AAV) vector-mediated delivery of NADH dehydrogenase subunit 4 (ND4) with mitochondrial targeting sequence, highlighting the differences in the vector design and the rationale behind their use in the allotopic transfer.
- |||||||||| Agamree (vamorolone) / Santhera
Trial primary completion date: A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) (clinicaltrials.gov) - Jun 7, 2023
P2, N=39, Recruiting,
Our study is the first to provide quantitative insights into VIDD-related myofibrillar remodeling unravelled by SHG imaging, suggesting that both VBP-15 and BGP-15 can effectively ameliorate the structure-related dysfunction in VIDD. Trial primary completion date: Aug 2024 --> Jan 2025
- |||||||||| vamorolone (VBP15) / Santhera
Undergraduate research-based education for identifying new treatment options for cystic fibrosis (Poster Area) - May 9, 2023 - Abstract #ECFS2023ECFS_475;
The students investigated the effects of VBP15 on intra- and extracellular MUC5AC and MUC5B expression, cell morphology, and toxicity. This research-based educational concept enhances student learning and development of skills that future professionals need to tackle complex problems with creative thinking and interdisciplinary collaboration, and with this concept innovative research for CF can be initiated potentially leading to better treatment options for all CF patients.
- |||||||||| Journal: Perspectives on the advances in the pharmacotherapeutic management of Duchenne muscular dystrophy. (Pubmed Central) - Nov 5, 2022
Corticosteroids such as prednisone and deflazacort are routinely given to patients to treat inflammation, but their use is limited by the occurrence of side effects and a lack of standardized prescribing...Excessive cost is a barrier to patients receiving medications that have yet to have established efficacy. Additional therapies have the potential to help patients with DMD, although most are several years away from approval for patient use.
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