Neuromuscular Diseases
EUROPEAN PARTNER
An Introduction to Neuromuscular Diseases
Neuromuscular diseases, characterized by muscle weakness, encompass a broad range of conditions that impair the function of voluntary muscles e.g. amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), myasthenia gravis and spinal muscular atrophy (SMA). The treatment landscape for neuromuscular diseases has seen numerous developments. Patients with SMA have had access to a range of new therapies including novel SMN-targeted treatments, antisense oligonucleotides and virus-mediated gene therapy. Likewise for patients with MS, with the introduction of new disease-modifying agents.
Browse video highlights and short articles from the conference hub, providing insights into the latest updates from major conferences and peer-reviewed articles from the journal portfolio. This is complimented by a range of educational activities from our expert faculty, with patient outcomes at the forefront.
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What if your medical degree could launch more than a clinical career? In this candid and compelling read, Dr Jon Edelson shares his lessons for early-career clinicians ready to think beyond the bedside and explore the business of medicine.
Broadest approval to date in generalized myasthenia gravis The FDA approval of nipocalimab for generalized myasthenia gravis (gMG) introduces a targeted therapy within a validated class, offering the potential for durable disease control across the broadest patient population to date. ...
In this episode, we explore the future of continuing medical education (CME) with the team behind touchIME. Hannah Fisher and Matthew Goodwin share insights into global and US trends, the importance of patient inclusivity and how educational outcomes are evolving to better measure the direct impact of learning on clinical practice and patient care.
Brain Awareness Week, taking place from March 10-16, 2025, is a global campaign dedicated to fostering public enthusiasm and support for brain science, organised by the Dana Foundation. Each March, participants worldwide organize imaginative activities that highlight the wonders of the brain and the profound impact of neuroscience on our daily lives.
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare, autoimmune neurological disorder in which peripheral nerve demyelination typically results in weakness, impaired limb sensation, fatigue and pain.1–4 CIDP may adversely affect activities of daily living, with a substantial impact on functional ...
The phase 3b SMART study has shown that 83% of paediatric patients with spinal muscular atrophy (SMA) weighing between 8.5 and 21 kg achieved significant motor improvements following treatment with intravenous onasemnogene abeparvovec in a recent article published in Neurology.
In this episode, we’re joined by Bradley Love, Professor of Cognitive and Decision Sciences at UCL, ELLIS fellow, and creator of BrainGPT. We discuss how this large language model is poised to assist researchers in advancing their work.
Duchenne muscular dystrophy (DMD) is an X-linked recessive, progressive and universally fatal disease in the spectrum of dystrophinopathies,1Â with an incidence of 21.4 patients in 100,000 live male births worldwide.2,3 Historically, patients with DMD would lose ambulation by the age of 10 due ...
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