Publications by authors named "Petra W Duda"
Article Synopsis
- A 12-week study showed that zilucoplan is effective in treating patients with generalized myasthenia gravis (gMG) who have acetylcholine receptor autoantibodies, but data beyond 12 weeks is limited.
- The study used a Bayesian model-informed analysis to predict zilucoplan's effects versus a control for up to 24 weeks, incorporating real-world and clinical trial data.
- Results showed significant improvement in the MG-ADL score for zilucoplan at 24 weeks, with a very high probability of a positive treatment effect compared to the control group.
Article Synopsis
- Generalized myasthenia gravis (gMG) is a chronic disease that significantly affects patients' quality of life and highlights the need for better treatments.
- The ongoing phase III study evaluates the long-term safety and effectiveness of the drug zilucoplan in patients with acetylcholine receptor autoantibody-positive gMG.
- Results from 200 patients showed that a significant majority experienced treatment-emergent adverse events, but those receiving zilucoplan showed continued improvements in daily living activities over 60 weeks, especially after switching from a placebo.
Article Synopsis
- The article DOI: 10.3389/fimmu.2023.1213920 has been updated to correct previous information.
- The correction addresses inaccuracies in data or findings presented in the original publication.
- Readers are encouraged to refer to the updated version for accurate and reliable information.
Article Synopsis
- The complement system plays a crucial role in the immune response, and its abnormal activation can lead to various diseases; zilucoplan, a macrocyclic peptide, effectively inhibits the activation of complement component C5.
- Research methods used to study zilucoplan's effectiveness included surface plasmon resonance, hemolysis assays, and ELISA to analyze its binding properties and impact on C5 activation, particularly for clinical variants with R885 polymorphisms.
- Results showed that zilucoplan binds strongly to C5, prevents its cleavage and the formation of the cytolytic membrane attack complex, and works effectively against C5 variants that do not respond to other treatments, indicating its potential for rapid clinical application.
Article Synopsis
- Generalised myasthenia gravis is a rare and difficult-to-manage disease that has a high treatment burden, highlighting the need for better therapies; zilucoplan, a self-administered treatment, was tested in this context.
- The RAISE trial was a phase 3 study conducted across 75 sites in multiple countries, comparing zilucoplan with a placebo in patients diagnosed with AChR-positive myasthenia gravis over a 12-week period.
- Results indicated that patients receiving zilucoplan experienced a significant reduction in their MG-ADL scores compared to those on placebo, suggesting it may be an effective treatment option.
Article Synopsis
- Immune-mediated necrotizing myopathy (IMNM) is an autoimmune condition causing muscle weakness and high creatine kinase levels, characterized by specific autoantibodies, with no approved treatments currently available.
- A study named IMNM01 was conducted to evaluate the drug zilucoplan, targeting complement C5, as a potential treatment for adults with anti-HMGCR or anti-SRP positive IMNM.
- Results showed no significant difference in muscle enzyme levels after eight weeks of treatment with zilucoplan compared to placebo, indicating that the drug did not lead to clinically meaningful improvements.
Introduction/aims: We studied the progression of myasthenia gravis (MG) disease burden and medication adjustment among MG Patient Registry participants.
Methods: Participants diagnosed with MG (age ≥18 years), registered between July 1, 2013 and July 31, 2018 and completing both 6- and 12-month follow-up surveys, were included in this investigation. Participants were grouped into high-burden (Myasthenia Gravis Activity of Daily Living scale [MG-ADL] score ≥6) and low-burden (MG-ADL <6) groups based on MG-ADL scores at enrollment.
Article Synopsis
- * Research is focusing on the complement cascade's role in gMG, leading to the investigation of a new treatment called zilucoplan, which specifically inhibits a component of the complement system to reduce muscle weakness.
- * In clinical trials, zilucoplan has shown promising results in inhibiting complement activity, indicating potential as a new treatment option for patients with gMG, subject to further phase 3 studies for efficacy and safety.
Introduction: Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Activities of Daily Living (MG-ADL) scales were compared using the data from the Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy (MGTX) study.
Methods: Correlation between QMG and MG-ADL raw and change-from-baseline scores was calculated every 3 months for 60 months based on treatment groups and minimal manifestation status (MMS).
Results: QMG and MG-ADL change-from-baseline scores correlated significantly, with increasing strength of correlation over time, in both treatment groups.
Article Synopsis
- Many patients with generalized myasthenia gravis (gMG) experience significant disability and need better therapies that are well-tolerated to improve their quality of life.
- A phase 2 clinical trial was conducted to assess the effects of zilucoplan, a self-administered injectable treatment, on patients with moderate to severe gMG.
- The study included 44 participants and compared the effectiveness of zilucoplan against a placebo over 12 weeks, focusing on changes in their disease symptoms and daily functioning.
Introduction: The Myasthenia Gravis Patient Registry (MGR) is a voluntary, patient-submitted database dedicated to improve understanding of care/burden of myasthenia gravis (MG).
Methods: In this study we present analyses of baseline records through July 2017 (n = 1140) containing data on the MG-Activities of Daily Living (MG-ADL) and the MG 15-item Quality of Life (MG-QOL15) instruments, two validated scales assessing quality of life in MG patients at sign-up into the MGR.
Results: Most registrants reported moderate to severe impairment of health-related quality of life, with a median MG-ADL score of 6 and a median MG-QOL15 score of 21.
Background: Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle loss and premature death, most commonly from respiratory or cardiac failure. DMD is primarily caused by whole exon deletions, resulting in a shift of the dystrophin mRNA reading frame that prevents production of functional dystrophin protein. Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO), is designed to skip exon 51, restore the reading frame, and induce production of internally shortened dystrophin in patients with mutations amenable to such treatment.
View Article and Find Full Text PDFImmune-mediated peripheral neuropathies are more frequent in aged populations. Equally, underlying diseases such as vasculitis and paraproteinemia are more prevalent in the elderly. Accumulating evidence is linking the aging process of the immune system, immunosenescence, to the susceptibility of older individuals for paraproteinemic, vasculitic and inflammatory demyelinating neuropathies.
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