Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study

The Lancet Neurology, Год журнала: 2023, Номер 22(5), С. 383 - 394

Опубликована: Апрель 13, 2023

Язык: Английский

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Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

European Journal of Neurology, Год журнала: 2024, Номер 31(5)

Опубликована: Фев. 6, 2024

Generalized myasthenia gravis (MG) with antibodies against the acetylcholine receptor is a chronic disease causing muscle weakness. Access to novel treatments warrants authoritative treatment recommendations. The Nordic countries have similar, comprehensive health systems, mandatory registers, and extensive MG research.

Язык: Английский

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Expert consensus recommendations for improving and standardising the assessment of patients with generalised myasthenia gravis

European Journal of Neurology, Год журнала: 2024, Номер 31(7)

Опубликована: Март 24, 2024

Regular and consistent disease assessment could provide a clearer picture of burden in generalised myasthenia gravis (gMG) improve patient care; however, the use tools practice lacks standardisation. This modified Delphi approach was taken to review current evidence on tool gMG develop expert-derived consensus recommendations for good practice.

Язык: Английский

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Inclusion in neurological research: empowering people living with neurological diseases

Nature Reviews Neurology, Год журнала: 2025, Номер unknown

Опубликована: Янв. 7, 2025

Язык: Английский

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Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient’s Perspective: A Qualitative Study

Neurology and Therapy, Год журнала: 2022, Номер 12(1), С. 107 - 128

Опубликована: Ноя. 2, 2022

Patients with generalized myasthenia gravis (gMG) experience functional impairment due to MG symptoms. This study aimed assess, from the patient perspective, symptoms, impacts, and treatment goals of individuals diagnosed gMG. Semi-structured, in-depth concept-elicitation interviews were conducted 28 gMG in United States. Participants reported symptoms that affected many body regions functions, an average 16 per participant. The most frequently eyelid drooping (93%), physical fatigue (89%), affecting legs (82%), difficulty breathing holding head up (82%). Nearly all participants (96%) fluctuations severity. Participants' bothersome blurry/double vision (43%), difficulties (36%), all-over swallowing problems (29%). Impacts on functioning included inability participate hobbies/sports, need for increased planning, performing activities daily living. All emotional impacts their work finances. Their reduced weakness, improved symptom stability, minimization impact particular impact. fluctuating unpredictable nature was found have a substantial patients' emotional, social, economic well-being. management suggest greater focus is needed help them quickly resume normal lifestyle by achieving stability. are difficult measure, but it important consider fluctuation as well ongoing symptomatology when making decisions, recognize uncontrolled patients, partners/caregivers, family/friends. These factors often not reflected burden/cost-of-illness studies. aim this understand—from patient's point view—the range they experience, which bother most, greatest everyday life, goals. Researchers asked these questions one-on-one people US who Study living come go, sometimes severe, lead life. (reported 93% participants), bothered patients trouble functioning, hard increasing amount planning conduct lives, hindering ability do day-to-day (like driving, eating, bathing). emotionally, impacted financial reducing more stable, particularly impacts. responses reveal extensive effects lives highlight stability especially

Язык: Английский

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Refocusing generalized myasthenia gravis: Patient burden, disease profiles, and the role of evolving therapy

European Journal of Neurology, Год журнала: 2023, Номер 31(6)

Опубликована: Дек. 20, 2023

Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due an unpredictable disease course, frequent fluctuations, and varying response therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity reevaluate how this is classified, assessed, managed identify ways improve the care patients with gMG.

Язык: Английский

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Patient authorship of medical research publications: An evolution, revolution, and solution?

Learned Publishing, Год журнала: 2024, Номер 37(3)

Опубликована: Апрель 30, 2024

Key points Patient authorship is a new topic in medical publishing, with the number of patient‐authored publications growing rapidly. Publication stakeholders (e.g., editors, publishers, funders, researchers) should be aware legitimacy patient authorship. Practical actions can taken by different publication to encourage and support authorship—ethically effectively. contribute enhancing diversity, equity, inclusion publishing. Research on could strengthened consensus tagging practices affiliation terms) use artificial intelligence. A plain language summary this article GRIPP2 form for reporting involvement are available as supplementary information.

Язык: Английский

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Recommendations for the management of myasthenia gravis in Belgium

Acta Neurologica Belgica, Год журнала: 2024, Номер 124(4), С. 1371 - 1383

Опубликована: Апрель 22, 2024

Abstract International guidelines on the treatment of myasthenia gravis (MG) have been published but are not tailored to Belgian situation. This publication presents recommendations from a group MG experts for practical management in Belgium. It includes adult patients with generalized (gMG) or ocular (oMG). Depending MG-related antibody sequence is suggested therapies that can be added if goal achieved. Selection treatments was based level evidence efficacy, registration and reimbursement status Belgium, common daily practice personal views experiences authors. The paper reflects situation February 2024. In addition considerations, other relevant aspects addressed, including comorbidities, drugs aggravating disease symptoms, pregnancy, vaccination. As many new might potentially come market, realistic future perspective impact these clinical given. conclusion, intend guide neurologists treating

Язык: Английский

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Rozanolixizumab in generalized myasthenia gravis: Pooled analysis of the Phase 3 MycarinG study and two open-label extensions

Journal of Neuromuscular Diseases, Год журнала: 2025, Номер unknown

Опубликована: Март 4, 2025

Background: Myasthenia gravis (MG) is a chronic autoimmune disease causing fluctuating muscle weakness. The MycarinG study showed that rozanolixizumab, neonatal Fc receptor inhibitor, provided clinically meaningful improvements in MG outcomes patients with acetylcholine (AChR) and muscle-specific tyrosine kinase (MuSK) autoantibody-positive generalized (gMG). Objective: We assessed efficacy safety of 6-week rozanolixizumab treatment cycles gMG. Methods: Following MycarinG, eligible enrolled the open-label extension Phase 3 studies MG0004 (NCT04124965) to receive up 52 weekly infusions or MG0007 (NCT04650854) 6 (initiated on symptom worsening at investigators’ discretion). To assess effect repeated cyclical treatment, data were pooled across (first weeks) (interim analysis). Efficacy endpoints included change from baseline Gravis Activities Daily Living (MG-ADL), Composite (MGC) Quantitative (QMG) who received ≥2 symptom-driven cycles. Treatment-emergent adverse events (TEAEs) ≥1 cycle had an (up to) 8-week follow-up period. Results: At cut-off (July 8, 2022), 188/196 (95.9%) period (primary pool; MycarinG/MG0007) 127 (64.8%) MycarinG/MG0004 [first weeks]/MG0007). Consistent MG-ADL, MGC QMG scores, high response rates, observed end first subsequent TEAEs experienced by 169/188 (89.9%) mostly mild moderate. did not increase Conclusions: Repeated resulted consistent, MG-specific acceptable profile, supporting as option for adults AChR MuSK

Язык: Английский

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Physician- and patient-reported perspectives on myasthenia gravis in Europe: a real-world survey

Orphanet Journal of Rare Diseases, Год журнала: 2023, Номер 18(1)

Опубликована: Июнь 29, 2023

Abstract Background Myasthenia gravis (MG) is a rare, chronic, debilitating, unpredictable, and potentially life-threatening neuromuscular disease. There lack of real-world data on disease management that could be used to further understand address unmet patient needs burden. We aimed provide comprehensive insights in the MG five European countries. Methods Data were collected using Adelphi Real World Disease Specific Programme™ MG, point-in-time survey physicians their patients with France, Germany, Italy, Spain, United Kingdom (UK). Physician- patient-reported clinical collected, including demographics, comorbidities, symptoms, history, treatments, healthcare resource utilization (HCRU), quality life outcomes. Results In total, 144 completed 778 record forms from March July 2020 UK, June September Italy Spain. Mean age at symptom onset was 47.7 years, mean time diagnosis 332.4 days (10.97 months). At diagnosis, 65.3% classified as Gravis Foundation America Class II or above. number symptoms reported per five, ocular myasthenia least 50% patients. completion, ptosis each still present more than Acetylcholinesterase inhibitors most commonly prescribed chronic treatments all Of 657 treated treatment survey, 62% continued experience moderate-to-severe symptoms. On average, 3.1 professionals (HCPs) involved management, 6.2 consultations made any HCP over last 12 months, 178 (22.9%) hospitalized months. Overall, HCRU similar across Conclusions Our findings demonstrated high burden despite current options for MG.

Язык: Английский

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