Rare Diseases
Rare Disease Report 2024
Diagnosing and Managing Duchenne Muscular Dystrophy: Tips for Practicing Clinicians
Healthcare providers should be familiar enough with Duchenne muscular dystrophy to provide timely diagnosis and early intervention as well as...
Rare Disease Report 2024
Promise for Disease-Modifying Therapies to Tame Huntington’s Disease
Much progress has been made in managing the symptoms of Huntington’s disease, but the real excitement lies in the development of DMARDs.
Rare Disease Report 2024
Newborn Screening Programs: What Do Clinicians Need to Know?
Rare Disease Report 2024
Genetic Testing for ALS, Now a Standard, Creates a Path Toward Individualized Care
Overall, there is a sense of progress in ALS. The hope is that clinical research is reaching a tipping point where targeted treatments may offer...
Latest News
Neurofibromatosis: What Affects Quality of Life Most?
“To develop effective therapeutics, meaningful clinical outcomes that are tied with improvement in QoL for persons with NF1 must be clearly...
From the Journals
Sex-Related Differences Found in IgG4-Related Disease Epidemiology
A recent study highlighted sex-based differences in manifestations of IgG4-related disease.
Conference Coverage
Upadacitinib Proves Successful in First JAK Inhibitor Trial for Giant Cell Arteritis
Results from the SELECT-GCA study showed that upadacitinib (Rinvoq) induced significant and sustained remission in people with new-onset or...
Hitting a Nerve
Knowing My Limits
In the daily trenches of general neurology, one can only be on top of so much. You have to prioritize the things you’re most likely to see and...
Latest News
Inebilizumab ‘MITIGATES’ Flare Risk in IgG4-Related Disease
Inebilizumab-cdon reduced the risk for flares by 87% in patients with IgG4-RD, the MITIGATE study showed.
Opinion
An 8-year-old girl presented with papules on her bilateral eyelid margins
Papules were slightly itchy but otherwise asymptomatic.
From the Journals
Diagnosing Giant Cell Arteritis Using Ultrasound First Proves Accurate, Avoids Biopsy in Many Cases
In a prospective study, all patients diagnosed with giant cell arteritis using ultrasound maintained their diagnosis for up to 2 years of follow-...