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. ...

Myasthenia gravis (MG) is an autoimmune disorder where antibodies disrupt the neuromuscular junction, causing muscle weakness that worsens with activity. MG subgroups are based on muscle weakness location, age, antibody type, and thymus pathology, with some patients experiencing severe, treatment-resistant symptoms. Biomarkers can indicate prognosis. First-line treatments include pyridostigmine for symptomatic relief and immunosuppressants like prednisolone and azathioprine. Thymectomy is recommended for certain patients. Second-line treatments include mycophenolate, rituximab, and others, with new therapies like complement and FcRn inhibitors showing promise. Intravenous immunoglobulin and plasma exchange are used for acute exacerbations. Supportive therapy, including adapted exercise, is crucial. In refractory cases, comorbidities and diagnosis accuracy should be reconsidered.

Neuromyelitis optica spectrum disorder (NMOSD) is a rare inflammatory autoimmune disease of the central nervous system (CNS) with a worldwide distribution.1 The first clinical description of NMOSD was made a century ago by Devic and Gault, who documented patients with ...

Duchenne muscular dystrophy (DMD) is an X-linked recessive, progressive and universally fatal disease in the spectrum of dystrophinopathies,1 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 ...

Case study Patient information A 42-year-old woman presented in the emergency department with acute onset whole-body myoclonic jerks for 1 day. On enquiry, the patient’s parents advised that she had a history of depression over the past 15 years. Intermittently, family ...

Acute disseminated encephalomyelitis (ADEM), first characterized in 1931,1 is a non-specific clinical syndrome of polyfocal central nervous system (CNS) inflammatory demyelination; it is characterized by encephalopathy and large, poorly demarcated cerebral white matter lesions.2,3 Myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOGAD), ...

Background for molecular-based therapy for spinal muscular atrophy Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease and the most common cause of infant death worldwide, with an incidence of 1:10,000 live births and carrier frequency of 1:50.1 This disease, caused ...

Myasthenia gravis (MG) represents the major and most frequent primary disorder of the neuromuscular junction. Clinical hallmarks are variable and include exercise-induced weakness involving extraocular, bulbar, limb and/or axial muscles.1 Respiratory involvement, characterized by orthopnoea or dyspnoea with exertion, ...

Infection of the central nervous system (CNS) due to cytomegalovirus (CMV) is rare, as it typically occurrs in immunocompromised patients and rarely affects those who are immunocompetent.1 Brain infection – manifested by meningoencephalitis – is much more common than infection of the ...