Publications by authors named "R Mantegazza"
Objectives: This post-hoc analysis evaluates the long-term efficacy of efgartigimod versus placebo in adult patients with generalized myasthenia gravis (gMG) with acetylcholine-receptor autoantibodies (AChR-Ab+), based on data from the ADAPT RCT and its open-label extension ADAPT+.
Methods: Changes from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores were assessed by treatment group over the ADAPT (up to 20 weeks) and ADAPT+ time horizon (extended to 64 weeks for efgartigimod group patients). Response to treatment was defined as 5-point reduction in QMG or 3-point reduction in MG-ADL vs.
Article Synopsis
- Myasthenia gravis (MG) is an autoimmune disorder that leads to muscle weakness and fatigue due to disrupted neuromuscular transmission caused by antibodies.
- The disease's development is influenced by multiple genetic and environmental factors, and while there has been progress in understanding the immune system's role in MG, the exact causes are still unclear.
- This article focuses on the importance of non-coding RNAs, particularly microRNAs and long non-coding RNAs, in MG, discussing their potential for therapeutic use and as biomarkers for personalized medicine.
Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study.
Ther Adv Neurol Disord
September 2024
Article Synopsis
- The study investigates the effectiveness and safety of rozanolixizumab in treating patients with muscle-specific tyrosine kinase (MuSK) autoantibody-positive generalized myasthenia gravis (gMG), which is a severe form of the condition.
- Conducted as a randomized, double-blind, placebo-controlled phase III trial, it involved participants aged 18 and older who were randomly assigned to receive either rozanolixizumab (at two different dosages) or a placebo over 6 weeks.
- Results showed significant improvements in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scores for both doses of rozanolixizumab compared to placebo, with manageable side effects reported;
ADAPT-SC (NCT04735432) was designed to evaluate noninferiority of subcutaneous (SC) efgartigimod PH20 to intravenous (IV) efgartigimod in participants with generalized myasthenia gravis (gMG). ADAPT-SC+ (NCT04818671) is an open-label extension study designed to assess long-term safety, tolerability, and efficacy of efgartigimod PH20 SC. Adult participants in ADAPT-SC were randomly assigned to receive a treatment cycle of 4 once-weekly administrations of efgartigimod PH20 SC 1000 mg or efgartigimod IV 10 mg/kg, followed by 7 weeks of follow-up.
View Article and Find Full Text PDFIntroduction/aims: Fatigue (subjective perception) and fatigability (objective motor performance worsening) are relevant aspects of disability in individuals with spinal muscular atrophy (SMA). The effect of nusinersen on fatigability in SMA patients has been investigated with conflicting results. We aimed to evaluate this in adult with SMA3.
View Article and Find Full Text PDF