Search Medical Journals & Research Articles

[Comment] The optimisation of migraine prevention: an urgent need

Migraine is a prevalent and disabling neurological condition that imposes a substantial burden on individuals and health systems, underscoring the need for effective preventive strategies. In their Review in The Lancet Neurology,1 Daniele Martinelli and colleagues highlight the latest treatment frameworks and research in preventing migraine. Their Review arrives at a pivotal moment in migraine management, as a growing body of evidence now supports the use of migraine-specific treatments, such as calcitonin gene-related peptide (CGRP) inhibitors, over traditional generic preventive medications including anticonvulsants, antihypertensives, and antidepressants.

[Comment] When Alzheimer's disease prevention becomes policy: regulatory speed, commercialisation, and risk

As Alzheimer's disease research focuses on earlier and asymptomatic stages, and as biomarker technologies advance, attention has turned to the feasibility of preventing cognitive impairment rather than treating established dementia. In their Policy View,1 Eric Reiman and colleagues argue that the Alzheimer's disease research, regulatory, and care community, including investigators, regulators, payers, industry, and health systems, should urgently prepare for the imminent prevention of clinical Alzheimer's disease.

[Comment] A step towards antiepileptogenic therapies for post-stroke epilepsy

Seizures are a recognised complication of both ischaemic stroke and intracranial haemorrhage, and around 6% of patients develop post-stroke epilepsy, the leading cause of late-onset epilepsy.1 Although many patients with post-stroke epilepsy respond to antiseizure medication, a recent meta-analysis showed increased rates of long-term disability, recurrent stroke, dementia, and all-cause mortality in patients with post-stroke epilepsy compared with those without post-stroke epilepsy.2 Post-stroke epilepsy is thought to arise from insults disrupting local excitatory and inhibitory cortical activity, leading to hyperexcitability, with suggested roles for neuroinflammation and iron-mediated oxidative stress.

[Comment] Broad lessons from negative trials in rare diseases

Negative trials are often perceived as setbacks, but they can be among the most instructive and essential for recalibrating assumptions in rapidly evolving fields such as gene therapy.1 Negative trials can prompt a cautious re-evaluation of surrogate endpoints, trial design, timing of intervention, and the interpretation of early-phase data, including reliance on historical controls. In The Lancet Neurology, Francesco Muntoni and colleagues report the negative results of the CIFFREO study,2 a large, randomised, double-blind, placebo-controlled, phase 3 trial evaluating fordadistrogene movaparvovec, an adeno-associated virus 9 (AAV9)-based mini-dystrophin gene therapy, in ambulant boys with Duchenne muscular dystrophy.

[Comment] Intensive rehabilitation after subacute spinal cord injury: more is not always better

Patients who have acute spinal cord injury face substantial neurological deficits and impairments in activities of daily living, yet they exhibit remarkable motivation to reclaim lost functions. Dedicated spinal cord injury centres allocate rehabilitation resources on the basis of treatment goals, medical stability, and the availability of necessary resources, resulting in structured schedules that encompass mobilisation, wheelchair skills training, and a multitude of targeted therapies aimed at improving sensorimotor function in both the upper and lower extremities.

[Editorial] Stopping Alzheimer's disease before symptoms start

Dementia due to Alzheimer's disease manifests years after the initial pathological changes in the brain. In fact, the deposition of amyloid plaques, and the subsequent tangles and neurodegeneration, can remain clinically silent for decades. This period presents a valuable opportunity for pharmacological treatments aimed at halting or delaying clinical manifestations. A Policy View in this issue explores initiatives that could soon facilitate the implementation of such treatments, at least in the USA.

Efgartigimod for Fetal Acetylcholine Receptor Antibody–Related Disorder

Use of off-label efgartigimod (a neonatal Fc receptor blocker) for fetal acetylcholine receptor antibody–related disorder during a pregnancy stabilized maternal antibodies and led to normal fetal development and a healthy birth.

A 12,000-Year-Old Case of NPR2-Related Acromesomelic Dysplasia

Rare disease in an adolescent who lived more than 12,000 years ago, along with her burial embrace by a first-degree relative, may reflect the importance of care for survival. Ancient DNA analysis led to a genetic diagnosis.

Majocchi’s Granuloma

A high-school wrestler presented with an itchy rash that had worsened despite topical antifungal and glucocorticoid treatment. A circular, scaly plaque with papules, nodules, and pustules was seen on his calf.

Shiga Toxin–Producing Escherichia coli Daycare Outbreak

This cohort study describes health care resource utilization, clinical outcomes, and outcomes associated with daily laboratory monitoring for thrombotic microangiopathy in a large, single-source Shiga toxin–producing Escherichia coli outbreak in Canadian daycare centers.

The Things We Carry

The ID, the pen, the epinephrine, the scalpel are the nonnegotiables, the necessities for any hospital shift. But they are not the only things a physician carries, and certainly not the most burdensome.

Sudden Cardiac Arrest in Athletes

Sudden cardiac arrest in athletes may be attributable to cardiac and noncardiac causes. With diagnosis and treatment, a return to play may be reasonable. Prevention, emergency planning, and shared decision making are key.

Pneumocystis jirovecii Pneumonia

A 60-year-old man with recently diagnosed HIV infection presented with a 10-day history of fever, dry cough, and shortness of breath. A CT scan of the chest showed diffuse nodules and ground-glass opacities.

LVAD

When a man ends up in the ED after having severed the driveline of his left ventricular assist device, the medical student on his team learns that sometimes a life saved is not the life the patient wants to live.

Primary Measles Infection

A previously healthy man presented with a 5-day history of fever, nausea, and vomiting and a 2-day history of an itchy rash. Examination showed an exanthem consisting of minute macules and subtly elevated papules.